Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2006-03

AUTHORS

Catherine S Manno, Glenn F Pierce, Valder R Arruda, Bertil Glader, Margaret Ragni, John J E Rasko, Margareth C Ozelo, Keith Hoots, Philip Blatt, Barbara Konkle, Michael Dake, Robin Kaye, Mahmood Razavi, Albert Zajko, James Zehnder, Pradip Rustagi, Hiroyuki Nakai, Amy Chew, Debra Leonard, J Fraser Wright, Ruth R Lessard, Jürg M Sommer, Michael Tigges, Denise Sabatino, Alvin Luk, Haiyan Jiang, Federico Mingozzi, Linda Couto, Hildegund C Ertl, Katherine A High, Mark A Kay

ABSTRACT

We have previously shown that a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic levels of F.IX in dogs with severe hemophilia B. We carried out a phase 1/2 dose-escalation clinical study to extend this approach to humans with severe hemophilia B. rAAV-2 vector expressing human F.IX was infused through the hepatic artery into seven subjects. The data show that: (i) vector infusion at doses up to 2 x 10(12) vg/kg was not associated with acute or long-lasting toxicity; (ii) therapeutic levels of F.IX were achieved at the highest dose tested; (iii) duration of expression at therapeutic levels was limited to a period of approximately 8 weeks; (iv) a gradual decline in F.IX was accompanied by a transient asymptomatic elevation of liver transaminases that resolved without treatment. Further studies suggested that destruction of transduced hepatocytes by cell-mediated immunity targeting antigens of the AAV capsid caused both the decline in F.IX and the transient transaminitis. We conclude that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels of F.IX, but that future studies in humans may require immunomodulation to achieve long-term expression. More... »

PAGES

342-347

Journal

TITLE

Nature Medicine

ISSUE

3

VOLUME

12

Related Patents

  • Methods And Compositions For Treating Hemophilia B
  • Tolerogenic Synthetic Nanocarriers For Inducing Regulatory B Cells
  • Method For Providing Polymeric Synthetic Nanocarriers For Generating Antigen-Specific Tolerance Immune Responses
  • Methods And Compositions For Attenuating Exon Skipping Anti-Viral Transfer Vector Immune Responses
  • Raav-Based Compositions And Methods
  • Optimized Liver-Specific Expression Systems For Fviii And Fix
  • Systemic Delivery And Regulated Expression Of Paracrine Genes For Cardiovascular Diseases And Other Conditions
  • Aav Capsid Proteins For Nucleic Acid Transfer
  • Raav Vector Compositions, Methods For Targeting Vascular Endothelial Cells And Use In Treatment Of Type I Diabetes
  • Improved Baculovirus Expression Systems
  • Systemic Delivery And Regulated Expression Of Paracrine Genes For Cardiovascular Diseases And Other Conditions
  • Treatment Of Ocular Neovascularization Using Anti-Vegf Proteins
  • Aav's And Uses Thereof
  • Compositions And Methods For Enhanced Gene Expression In Cone Cells
  • Isolation Of Novel Aav's And Uses Thereof
  • Delivery Of Proteins Using Adeno-Associated Virus (Aav) Vectors
  • Liver Organoid, Uses Thereof And Culture Method For Obtaining Them
  • Aav-Based Treatment Of Cholesterol-Related Disorders
  • Raav Vector Compositions Having Tyrosine-Modified Capsid Proteins And Methods For Use
  • Methods And Compositions For Treating Hemophilia
  • Methods And Compositions For Treating Hemophilia B
  • Raav-Based Compositions And Methods For Treating Alpha-1 Anti-Trypsin Deficiencies
  • Method For Introducing An Ultraviolet Light Activated Viral Vector Into The Spinal Column
  • Gene Therapy Composition For Use In Diabetes Treatment
  • Treatment Of Ocular Neovascularization Using Anti-Vegf Proteins
  • Method Of Reducing Titers Of Antibodies Specific For A Therapeutic Agent
  • Adeno-Associated Virus With Site-Directed Mutagenesis And Site-Directed Modification, And Preparation Method And Application Therefor
  • Cns Targeting Aav Vectors And Methods Of Use Thereof
  • Aav's And Uses Thereof
  • Delivery Of Proteins Using Adeno-Associated Virus (Aav) Vectors
  • Hyper-Active Factor Ix Vectors For Liver-Directed Gene Therapy Of Hemophilia 'B' And Methods And Use Thereof
  • Gene Therapy Composition For Use In Diabetes Treatment
  • Synergistic Combination Of Neuronal Viability Factors And Uses Thereof
  • Culture Medium For Epithelial Stem Cells And Organoids Comprising The Stem Cells
  • Isolation Of Novel Aav's And Uses Thereof
  • Multicistronic Expression Constructs
  • Methods And Compositions For Genome Engineering
  • Methods And Compositions For Attenuating Gene Expression Modulating Anti-Viral Transfer Vector Immune Responses
  • Light Activated Gene Transduction Using Long Wavelength Ultraviolet Light For Cell Targeted Gene Delivery
  • Cns Targeting Aav Vectors And Methods Of Use Thereof
  • Compositions And Methods For Detection And Modulation Of T Cell Mediated Immune Responses Against Viral Vectors Utilized For Gene Therapy
  • Aav's And Uses Thereof
  • Tolerogenic Synthetic Nanocarriers For Antigen-Specific Deletion Of T Effector Cells
  • Tolerogenic Synthetic Nanocarriers For Generating Cd8+ Regulatory T Cells
  • Recombinant Human Factor Ix And Use Thereof
  • Novel Recombinant Adeno-Associated Virus Capsids Resistant To Pre-Existing Human Neutralizing Antibodies
  • Aav Capsid Proteins For Nucleic Acid Transfer
  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/nm1358

    DOI

    http://dx.doi.org/10.1038/nm1358

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1012134968

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/16474400


    Indexing Status Check whether this publication has been indexed by Scopus and Web Of Science using the SN Indexing Status Tool
    Incoming Citations Browse incoming citations for this publication using opencitations.net

    JSON-LD is the canonical representation for SciGraph data.

    TIP: You can open this SciGraph record using an external JSON-LD service: JSON-LD Playground Google SDTT

    [
      {
        "@context": "https://springernature.github.io/scigraph/jsonld/sgcontext.json", 
        "about": [
          {
            "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/1103", 
            "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
            "name": "Clinical Sciences", 
            "type": "DefinedTerm"
          }, 
          {
            "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/11", 
            "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
            "name": "Medical and Health Sciences", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Adult", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Amino Acid Sequence", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Animals", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Dependovirus", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Dogs", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Dose-Response Relationship, Drug", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Exons", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Factor IX", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Genetic Therapy", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Genetic Vectors", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Hemophilia A", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Humans", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Interferon-gamma", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Introns", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Liver", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Male", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Mice", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Middle Aged", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Molecular Sequence Data", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Monocytes", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Transduction, Genetic", 
            "type": "DefinedTerm"
          }
        ], 
        "author": [
          {
            "affiliation": {
              "alternateName": "University of Pennsylvania", 
              "id": "https://www.grid.ac/institutes/grid.25879.31", 
              "name": [
                "The Children's Hospital of Philadelphia, and", 
                "University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Manno", 
            "givenName": "Catherine S", 
            "id": "sg:person.01211363720.11", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01211363720.11"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "name": [
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Pierce", 
            "givenName": "Glenn F", 
            "id": "sg:person.016335301372.89", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.016335301372.89"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "University of Pennsylvania", 
              "id": "https://www.grid.ac/institutes/grid.25879.31", 
              "name": [
                "The Children's Hospital of Philadelphia, and", 
                "University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Arruda", 
            "givenName": "Valder R", 
            "id": "sg:person.01035106403.91", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01035106403.91"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Stanford University", 
              "id": "https://www.grid.ac/institutes/grid.168010.e", 
              "name": [
                "Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, California 94305-5208, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Glader", 
            "givenName": "Bertil", 
            "id": "sg:person.01363703525.35", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01363703525.35"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "University of Pittsburgh Medical Center", 
              "id": "https://www.grid.ac/institutes/grid.412689.0", 
              "name": [
                "University of Pittsburgh Medical Center, Hemophilia Center of Western Pennsylvania, 3636 Boulevard of the Allies, Pittsburgh, Pennsylvania 15213, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Ragni", 
            "givenName": "Margaret", 
            "id": "sg:person.0664442574.69", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0664442574.69"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Royal Prince Alfred Hospital", 
              "id": "https://www.grid.ac/institutes/grid.413249.9", 
              "name": [
                "Royal Prince Alfred Hospital, Centenary Institute of Cancer Medicine & Cell Biology, Locked Bag No 6, Newtown NSW 2042, Australia."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Rasko", 
            "givenName": "John J E", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "State University of Campinas", 
              "id": "https://www.grid.ac/institutes/grid.411087.b", 
              "name": [
                "University of Campinas, State University of Campinas, Caixa Postal 6198, CEP, 13083-970, Campinas-Sao Paolo, Brazil."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Ozelo", 
            "givenName": "Margareth C", 
            "id": "sg:person.0654065663.76", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0654065663.76"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "The University of Texas Health Science Center at Houston", 
              "id": "https://www.grid.ac/institutes/grid.267308.8", 
              "name": [
                "University of Texas Houston Health Science Center, 1515 Holcombe Boulevard, Houston, Texas 77030, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Hoots", 
            "givenName": "Keith", 
            "id": "sg:person.0662644741.32", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0662644741.32"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Christiana Hospital", 
              "id": "https://www.grid.ac/institutes/grid.414314.7", 
              "name": [
                "Christiana Care, Christiana Hospital, 4755 Ogletown-Stanton Road, Newark, Delaware 19713, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Blatt", 
            "givenName": "Philip", 
            "id": "sg:person.053412353.52", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.053412353.52"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "University of Pennsylvania", 
              "id": "https://www.grid.ac/institutes/grid.25879.31", 
              "name": [
                "University of Pennsylvania School of Medicine Presbyterian Medical Center, MAB, Suite 103, 39th & Market Street, Philadelphia, Pennsylvania 19104, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Konkle", 
            "givenName": "Barbara", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Stanford University", 
              "id": "https://www.grid.ac/institutes/grid.168010.e", 
              "name": [
                "Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, California 94305-5208, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Dake", 
            "givenName": "Michael", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "University of Pennsylvania", 
              "id": "https://www.grid.ac/institutes/grid.25879.31", 
              "name": [
                "The Children's Hospital of Philadelphia, and", 
                "University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Kaye", 
            "givenName": "Robin", 
            "id": "sg:person.01237547567.14", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01237547567.14"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Stanford University", 
              "id": "https://www.grid.ac/institutes/grid.168010.e", 
              "name": [
                "Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, California 94305-5208, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Razavi", 
            "givenName": "Mahmood", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "University of Pennsylvania", 
              "id": "https://www.grid.ac/institutes/grid.25879.31", 
              "name": [
                "University of Pennsylvania School of Medicine Presbyterian Medical Center, MAB, Suite 103, 39th & Market Street, Philadelphia, Pennsylvania 19104, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Zajko", 
            "givenName": "Albert", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Stanford University", 
              "id": "https://www.grid.ac/institutes/grid.168010.e", 
              "name": [
                "Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, California 94305-5208, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Zehnder", 
            "givenName": "James", 
            "type": "Person"
          }, 
          {
            "familyName": "Rustagi", 
            "givenName": "Pradip", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Stanford University", 
              "id": "https://www.grid.ac/institutes/grid.168010.e", 
              "name": [
                "Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, California 94305-5208, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Nakai", 
            "givenName": "Hiroyuki", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Children's Hospital of Philadelphia", 
              "id": "https://www.grid.ac/institutes/grid.239552.a", 
              "name": [
                "The Children's Hospital of Philadelphia, and", 
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Chew", 
            "givenName": "Amy", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Cornell University", 
              "id": "https://www.grid.ac/institutes/grid.5386.8", 
              "name": [
                "University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA.", 
                "Weill Medical College of Cornell University, 525 East 68th Street, New York, New York 10012, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Leonard", 
            "givenName": "Debra", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "name": [
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Wright", 
            "givenName": "J Fraser", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "name": [
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Lessard", 
            "givenName": "Ruth R", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "name": [
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Sommer", 
            "givenName": "J\u00fcrg M", 
            "id": "sg:person.01250056754.47", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01250056754.47"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "name": [
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Tigges", 
            "givenName": "Michael", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Children's Hospital of Philadelphia", 
              "id": "https://www.grid.ac/institutes/grid.239552.a", 
              "name": [
                "The Children's Hospital of Philadelphia, and"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Sabatino", 
            "givenName": "Denise", 
            "id": "sg:person.01001004070.50", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01001004070.50"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "name": [
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Luk", 
            "givenName": "Alvin", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "name": [
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Jiang", 
            "givenName": "Haiyan", 
            "id": "sg:person.01340662554.04", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01340662554.04"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Children's Hospital of Philadelphia", 
              "id": "https://www.grid.ac/institutes/grid.239552.a", 
              "name": [
                "The Children's Hospital of Philadelphia, and"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Mingozzi", 
            "givenName": "Federico", 
            "id": "sg:person.01145473703.03", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01145473703.03"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "name": [
                "Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Couto", 
            "givenName": "Linda", 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Wistar Institute", 
              "id": "https://www.grid.ac/institutes/grid.251075.4", 
              "name": [
                "The Children's Hospital of Philadelphia, and", 
                "Wistar Institute, 3601 Spruce Street, Philadelphia, Pennsylvania 19104, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Ertl", 
            "givenName": "Hildegund C", 
            "id": "sg:person.01200036420.15", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01200036420.15"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Howard Hughes Medical Institute", 
              "id": "https://www.grid.ac/institutes/grid.413575.1", 
              "name": [
                "The Children's Hospital of Philadelphia, and", 
                "University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA.", 
                "Howard Hughes Medical Institute, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "High", 
            "givenName": "Katherine A", 
            "id": "sg:person.01104637520.44", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01104637520.44"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Stanford University", 
              "id": "https://www.grid.ac/institutes/grid.168010.e", 
              "name": [
                "Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, California 94305-5208, USA."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Kay", 
            "givenName": "Mark A", 
            "id": "sg:person.01164322460.34", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01164322460.34"
            ], 
            "type": "Person"
          }
        ], 
        "citation": [
          {
            "id": "sg:pub.10.1038/ng0501-92", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1001946207", 
              "https://doi.org/10.1038/ng0501-92"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/ng0501-92", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1001946207", 
              "https://doi.org/10.1038/ng0501-92"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.smim.2004.02.007", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1002351699"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1182/blood-2002-10-3296", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1003619350"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/4743", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1005137439", 
              "https://doi.org/10.1038/4743"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/4743", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1005137439", 
              "https://doi.org/10.1038/4743"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/sj.gt.3301024", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1008888203", 
              "https://doi.org/10.1038/sj.gt.3301024"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/sj.gt.3301024", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1008888203", 
              "https://doi.org/10.1038/sj.gt.3301024"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/s0022-1759(01)00535-x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1010317193"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1128/jvi.78.24.13678-13686.2004", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1011327932"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1128/jvi.78.6.3110-3122.2004", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1013436925"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1074/jbc.270.10.5276", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1015848421"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1182/blood-2004-10-3867", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1018702261"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1182/blood.v99.8.2670", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1019564621"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1182/blood-2003-01-0292", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1021429034"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/73464", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1023348153", 
              "https://doi.org/10.1038/73464"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/73464", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1023348153", 
              "https://doi.org/10.1038/73464"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1006/mthe.2001.0491", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1023556799"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/s0022-1759(03)00226-6", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1026782649"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/s0022-1759(03)00226-6", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1026782649"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1182/blood-2004-07-2908", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1026834251"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/hep.1840200104", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1029401994"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1046/j.1365-2141.1998.00707.x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1035175583"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1046/j.1365-2141.1998.00707.x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1035175583"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1182/blood-2005-10-4035", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1035241251"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1182/blood-2002-02-0589", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1036180752"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1046/j.1365-2796.1997.130135000.x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1036948431"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1046/j.1365-2796.1997.130135000.x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1036948431"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/hep.510240201", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1038541144"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/4751", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1043560823", 
              "https://doi.org/10.1038/4751"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/4751", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1043560823", 
              "https://doi.org/10.1038/4751"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1006/mthe.2000.0031", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1045595102"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.ymthe.2005.02.021", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1049406390"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1006/mthe.2000.0075", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1051938287"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/s1525-0016(02)00019-9", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1053021243"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/s1525-0016(02)00019-9", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1053021243"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1089/104303404322886138", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1059202704"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://app.dimensions.ai/details/publication/pub.1083044374", 
            "type": "CreativeWork"
          }
        ], 
        "datePublished": "2006-03", 
        "datePublishedReg": "2006-03-01", 
        "description": "We have previously shown that a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic levels of F.IX in dogs with severe hemophilia B. We carried out a phase 1/2 dose-escalation clinical study to extend this approach to humans with severe hemophilia B. rAAV-2 vector expressing human F.IX was infused through the hepatic artery into seven subjects. The data show that: (i) vector infusion at doses up to 2 x 10(12) vg/kg was not associated with acute or long-lasting toxicity; (ii) therapeutic levels of F.IX were achieved at the highest dose tested; (iii) duration of expression at therapeutic levels was limited to a period of approximately 8 weeks; (iv) a gradual decline in F.IX was accompanied by a transient asymptomatic elevation of liver transaminases that resolved without treatment. Further studies suggested that destruction of transduced hepatocytes by cell-mediated immunity targeting antigens of the AAV capsid caused both the decline in F.IX and the transient transaminitis. We conclude that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels of F.IX, but that future studies in humans may require immunomodulation to achieve long-term expression.", 
        "genre": "research_article", 
        "id": "sg:pub.10.1038/nm1358", 
        "inLanguage": [
          "en"
        ], 
        "isAccessibleForFree": false, 
        "isFundedItemOf": [
          {
            "id": "sg:grant.2403943", 
            "type": "MonetaryGrant"
          }, 
          {
            "id": "sg:grant.2538929", 
            "type": "MonetaryGrant"
          }, 
          {
            "id": "sg:grant.2691684", 
            "type": "MonetaryGrant"
          }, 
          {
            "id": "sg:grant.2436938", 
            "type": "MonetaryGrant"
          }
        ], 
        "isPartOf": [
          {
            "id": "sg:journal.1113716", 
            "issn": [
              "1078-8956", 
              "1546-170X"
            ], 
            "name": "Nature Medicine", 
            "type": "Periodical"
          }, 
          {
            "issueNumber": "3", 
            "type": "PublicationIssue"
          }, 
          {
            "type": "PublicationVolume", 
            "volumeNumber": "12"
          }
        ], 
        "name": "Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response", 
        "pagination": "342-347", 
        "productId": [
          {
            "name": "readcube_id", 
            "type": "PropertyValue", 
            "value": [
              "39d086bbbdc0e24974c85dee6563d2011ec42a2dbae0dd6d83e6a9414f3e28a6"
            ]
          }, 
          {
            "name": "pubmed_id", 
            "type": "PropertyValue", 
            "value": [
              "16474400"
            ]
          }, 
          {
            "name": "nlm_unique_id", 
            "type": "PropertyValue", 
            "value": [
              "9502015"
            ]
          }, 
          {
            "name": "doi", 
            "type": "PropertyValue", 
            "value": [
              "10.1038/nm1358"
            ]
          }, 
          {
            "name": "dimensions_id", 
            "type": "PropertyValue", 
            "value": [
              "pub.1012134968"
            ]
          }
        ], 
        "sameAs": [
          "https://doi.org/10.1038/nm1358", 
          "https://app.dimensions.ai/details/publication/pub.1012134968"
        ], 
        "sdDataset": "articles", 
        "sdDatePublished": "2019-04-11T12:23", 
        "sdLicense": "https://scigraph.springernature.com/explorer/license/", 
        "sdPublisher": {
          "name": "Springer Nature - SN SciGraph project", 
          "type": "Organization"
        }, 
        "sdSource": "s3://com-uberresearch-data-dimensions-target-20181106-alternative/cleanup/v134/2549eaecd7973599484d7c17b260dba0a4ecb94b/merge/v9/a6c9fde33151104705d4d7ff012ea9563521a3ce/jats-lookup/v90/0000000362_0000000362/records_87091_00000000.jsonl", 
        "type": "ScholarlyArticle", 
        "url": "http://www.nature.com/articles/nm1358"
      }
    ]
     

    Download the RDF metadata as:  json-ld nt turtle xml License info

    HOW TO GET THIS DATA PROGRAMMATICALLY:

    JSON-LD is a popular format for linked data which is fully compatible with JSON.

    curl -H 'Accept: application/ld+json' 'https://scigraph.springernature.com/pub.10.1038/nm1358'

    N-Triples is a line-based linked data format ideal for batch operations.

    curl -H 'Accept: application/n-triples' 'https://scigraph.springernature.com/pub.10.1038/nm1358'

    Turtle is a human-readable linked data format.

    curl -H 'Accept: text/turtle' 'https://scigraph.springernature.com/pub.10.1038/nm1358'

    RDF/XML is a standard XML format for linked data.

    curl -H 'Accept: application/rdf+xml' 'https://scigraph.springernature.com/pub.10.1038/nm1358'


     

    This table displays all metadata directly associated to this object as RDF triples.

    499 TRIPLES      21 PREDICATES      79 URIs      42 LITERALS      30 BLANK NODES

    Subject Predicate Object
    1 sg:pub.10.1038/nm1358 schema:about N06deec89bf2146e587a8e48379171a96
    2 N092fd595ed334b7bbb6339633fee3e1a
    3 N2b019efbeb51422d82feefedc7a7df7f
    4 N2c6d9d00e72d4f9dad635fe6bf103987
    5 N30006e114d5a4d3dbe58649a45554f99
    6 N33186a76dc734346a5822f61cbc22b0e
    7 N567406145555485b941909b85d0c3a24
    8 N61b3200f92bb44d4961f810d36125015
    9 N765a55a1281a444eb6ae3ed93c622bbd
    10 N7deff6ecc0584fffa3f43dea6a1cc71a
    11 N8b1381bfa4e641cbaf1a614ec0f89128
    12 N8c06f3fa746b443597919b7e9a34eb34
    13 N9c92479ee23d46e49b870929c1bdf733
    14 Na756c2c92d17487d8ca4e9819dfb3d74
    15 Nb71cc538970c4037b4bf894c28aab69a
    16 Nbcf323d5cf4745d88886a890943c7d33
    17 Nbfbfbd0012e1420a85dc479e77de1d18
    18 Nc2f080b9b47345d28ec84e0513afdf43
    19 Nc30c62cc76654e05903c42a91cd23802
    20 Nd4bea0f936334169842c787535990ece
    21 Nd9596279389b42fbb1abff29429f9354
    22 anzsrc-for:11
    23 anzsrc-for:1103
    24 schema:author N6c98742eda9945b38c09ebbbd8812604
    25 schema:citation sg:pub.10.1038/4743
    26 sg:pub.10.1038/4751
    27 sg:pub.10.1038/73464
    28 sg:pub.10.1038/ng0501-92
    29 sg:pub.10.1038/sj.gt.3301024
    30 https://app.dimensions.ai/details/publication/pub.1083044374
    31 https://doi.org/10.1002/hep.1840200104
    32 https://doi.org/10.1002/hep.510240201
    33 https://doi.org/10.1006/mthe.2000.0031
    34 https://doi.org/10.1006/mthe.2000.0075
    35 https://doi.org/10.1006/mthe.2001.0491
    36 https://doi.org/10.1016/j.smim.2004.02.007
    37 https://doi.org/10.1016/j.ymthe.2005.02.021
    38 https://doi.org/10.1016/s0022-1759(01)00535-x
    39 https://doi.org/10.1016/s0022-1759(03)00226-6
    40 https://doi.org/10.1016/s1525-0016(02)00019-9
    41 https://doi.org/10.1046/j.1365-2141.1998.00707.x
    42 https://doi.org/10.1046/j.1365-2796.1997.130135000.x
    43 https://doi.org/10.1074/jbc.270.10.5276
    44 https://doi.org/10.1089/104303404322886138
    45 https://doi.org/10.1128/jvi.78.24.13678-13686.2004
    46 https://doi.org/10.1128/jvi.78.6.3110-3122.2004
    47 https://doi.org/10.1182/blood-2002-02-0589
    48 https://doi.org/10.1182/blood-2002-10-3296
    49 https://doi.org/10.1182/blood-2003-01-0292
    50 https://doi.org/10.1182/blood-2004-07-2908
    51 https://doi.org/10.1182/blood-2004-10-3867
    52 https://doi.org/10.1182/blood-2005-10-4035
    53 https://doi.org/10.1182/blood.v99.8.2670
    54 schema:datePublished 2006-03
    55 schema:datePublishedReg 2006-03-01
    56 schema:description We have previously shown that a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic levels of F.IX in dogs with severe hemophilia B. We carried out a phase 1/2 dose-escalation clinical study to extend this approach to humans with severe hemophilia B. rAAV-2 vector expressing human F.IX was infused through the hepatic artery into seven subjects. The data show that: (i) vector infusion at doses up to 2 x 10(12) vg/kg was not associated with acute or long-lasting toxicity; (ii) therapeutic levels of F.IX were achieved at the highest dose tested; (iii) duration of expression at therapeutic levels was limited to a period of approximately 8 weeks; (iv) a gradual decline in F.IX was accompanied by a transient asymptomatic elevation of liver transaminases that resolved without treatment. Further studies suggested that destruction of transduced hepatocytes by cell-mediated immunity targeting antigens of the AAV capsid caused both the decline in F.IX and the transient transaminitis. We conclude that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels of F.IX, but that future studies in humans may require immunomodulation to achieve long-term expression.
    57 schema:genre research_article
    58 schema:inLanguage en
    59 schema:isAccessibleForFree false
    60 schema:isPartOf N779aec2bc73246df8c4731a93f89417d
    61 N80381ebb431b44d1bf08281245fd0f97
    62 sg:journal.1113716
    63 schema:name Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    64 schema:pagination 342-347
    65 schema:productId N034312f042324b49b05705924b29ccd8
    66 N6becd2c2655c4eb4918b374e39989fc7
    67 N8ee92968deb743bb8e3306372dcd4285
    68 Nc563d5a798de4dde93f88978608445e9
    69 Nfc4e24a42cac499dbdbf088f475748d9
    70 schema:sameAs https://app.dimensions.ai/details/publication/pub.1012134968
    71 https://doi.org/10.1038/nm1358
    72 schema:sdDatePublished 2019-04-11T12:23
    73 schema:sdLicense https://scigraph.springernature.com/explorer/license/
    74 schema:sdPublisher Na5d1565ab5a848ae9e59e2c309b5ec40
    75 schema:url http://www.nature.com/articles/nm1358
    76 sgo:license sg:explorer/license/
    77 sgo:sdDataset articles
    78 rdf:type schema:ScholarlyArticle
    79 N02e8a29304284ce1a2f8ecbf1e3e9295 schema:affiliation N9ecf32ba32ad4031af8af9600fc5b5e0
    80 schema:familyName Tigges
    81 schema:givenName Michael
    82 rdf:type schema:Person
    83 N034312f042324b49b05705924b29ccd8 schema:name nlm_unique_id
    84 schema:value 9502015
    85 rdf:type schema:PropertyValue
    86 N03660bf9d5214abf8bc8f8ad1364243d schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    87 rdf:type schema:Organization
    88 N06deec89bf2146e587a8e48379171a96 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    89 schema:name Male
    90 rdf:type schema:DefinedTerm
    91 N092fd595ed334b7bbb6339633fee3e1a schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    92 schema:name Amino Acid Sequence
    93 rdf:type schema:DefinedTerm
    94 N130958372253465aab2cae790b98cbac rdf:first Nec25481284f540a48f483ff396e94299
    95 rdf:rest N9790f97a0d454e1bac4cf132b79b0a5b
    96 N177e6c0e415f4b8faee3197cd38fd93b rdf:first sg:person.0662644741.32
    97 rdf:rest N661dd04a19144f29abc277c78ee14b8d
    98 N1ecff20edf14416f97f37525974bd412 rdf:first sg:person.01164322460.34
    99 rdf:rest rdf:nil
    100 N1f9acc57c41b4a5192997b7cf1e6faeb rdf:first sg:person.0654065663.76
    101 rdf:rest N177e6c0e415f4b8faee3197cd38fd93b
    102 N20213d2581d2452cb6dd852951151c2f schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    103 rdf:type schema:Organization
    104 N27daaf305ad043f990c74dff86777876 schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    105 rdf:type schema:Organization
    106 N27dd3d22826844d9bf762ccbdbd5958b schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    107 rdf:type schema:Organization
    108 N2b019efbeb51422d82feefedc7a7df7f schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    109 schema:name Adult
    110 rdf:type schema:DefinedTerm
    111 N2c6d9d00e72d4f9dad635fe6bf103987 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    112 schema:name Monocytes
    113 rdf:type schema:DefinedTerm
    114 N30006e114d5a4d3dbe58649a45554f99 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    115 schema:name Humans
    116 rdf:type schema:DefinedTerm
    117 N3265ad48ecbf45af9df6dff280c10277 schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    118 rdf:type schema:Organization
    119 N33186a76dc734346a5822f61cbc22b0e schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    120 schema:name Genetic Therapy
    121 rdf:type schema:DefinedTerm
    122 N368b4b46861a4d45aed865328a9310cd rdf:first sg:person.01250056754.47
    123 rdf:rest Nfd809a5e9cde4ec1ada717ae15f47606
    124 N39d86dea0de443989baeacc6b47f1c22 schema:affiliation https://www.grid.ac/institutes/grid.168010.e
    125 schema:familyName Zehnder
    126 schema:givenName James
    127 rdf:type schema:Person
    128 N3c0fe3dc113941f4a82e192801d5fec5 rdf:first sg:person.0664442574.69
    129 rdf:rest Nb128647e60a64d0a8d43aad384babc6d
    130 N3ec354d491f748a59ba65c8ced79e8d1 rdf:first Nedb2686e2c794ae58e918ce8bbabcda0
    131 rdf:rest N43da7b2c6cdf4fa4b4e827c69103882c
    132 N43da7b2c6cdf4fa4b4e827c69103882c rdf:first sg:person.01340662554.04
    133 rdf:rest N85407bbcc929423abebac55f1e650c7b
    134 N4cf8a538e2664876b59cd8ed56f78817 rdf:first Nbac50b8927ae4213a9e96048d75956d0
    135 rdf:rest N91f7818d671a4945bd01ab9f66fd4866
    136 N4fe97376c69d4c91b0c3a0538505cfe2 schema:affiliation https://www.grid.ac/institutes/grid.25879.31
    137 schema:familyName Konkle
    138 schema:givenName Barbara
    139 rdf:type schema:Person
    140 N567406145555485b941909b85d0c3a24 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    141 schema:name Molecular Sequence Data
    142 rdf:type schema:DefinedTerm
    143 N57dee8906ee64bfd9d5c3419008f8b7d rdf:first Nf12b8de5a1164d9e8b8a22885dcacb5e
    144 rdf:rest N5f6967c0ff6a4891afe94928f0fce93d
    145 N5d84de8217444a06ad41a284a2511562 schema:affiliation https://www.grid.ac/institutes/grid.5386.8
    146 schema:familyName Leonard
    147 schema:givenName Debra
    148 rdf:type schema:Person
    149 N5f6967c0ff6a4891afe94928f0fce93d rdf:first N935a46d49c194800a88d4adbed0f2b07
    150 rdf:rest Nccd0c9d744d5485b81d5568afb9e78f3
    151 N61b3200f92bb44d4961f810d36125015 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    152 schema:name Dose-Response Relationship, Drug
    153 rdf:type schema:DefinedTerm
    154 N661dd04a19144f29abc277c78ee14b8d rdf:first sg:person.053412353.52
    155 rdf:rest N9579cbfd3ae146dab6cb0a46308e3cea
    156 N6ac3e0d435b44056b25a493a063e7f24 schema:affiliation N03660bf9d5214abf8bc8f8ad1364243d
    157 schema:familyName Lessard
    158 schema:givenName Ruth R
    159 rdf:type schema:Person
    160 N6becd2c2655c4eb4918b374e39989fc7 schema:name dimensions_id
    161 schema:value pub.1012134968
    162 rdf:type schema:PropertyValue
    163 N6c98742eda9945b38c09ebbbd8812604 rdf:first sg:person.01211363720.11
    164 rdf:rest Ne190f45d46eb424cb2ea3d253ad8c434
    165 N6e4565e720cc459299a040dc5f0a14bc rdf:first sg:person.01363703525.35
    166 rdf:rest N3c0fe3dc113941f4a82e192801d5fec5
    167 N6f64dd585fce4b69958a549d90dd23e4 schema:affiliation https://www.grid.ac/institutes/grid.25879.31
    168 schema:familyName Zajko
    169 schema:givenName Albert
    170 rdf:type schema:Person
    171 N73079fd8237444d1b9c018f7bfd2d6e7 schema:affiliation N3265ad48ecbf45af9df6dff280c10277
    172 schema:familyName Couto
    173 schema:givenName Linda
    174 rdf:type schema:Person
    175 N750a454bbbc64a49ac9246b19c8096eb schema:familyName Rustagi
    176 schema:givenName Pradip
    177 rdf:type schema:Person
    178 N765a55a1281a444eb6ae3ed93c622bbd schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    179 schema:name Mice
    180 rdf:type schema:DefinedTerm
    181 N779aec2bc73246df8c4731a93f89417d schema:issueNumber 3
    182 rdf:type schema:PublicationIssue
    183 N7deff6ecc0584fffa3f43dea6a1cc71a schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    184 schema:name Transduction, Genetic
    185 rdf:type schema:DefinedTerm
    186 N80381ebb431b44d1bf08281245fd0f97 schema:volumeNumber 12
    187 rdf:type schema:PublicationVolume
    188 N85407bbcc929423abebac55f1e650c7b rdf:first sg:person.01145473703.03
    189 rdf:rest Nbf85230f6d55459182214cc9768f4aa3
    190 N87f06356651c43cab9e37b15e5adeea5 rdf:first sg:person.01001004070.50
    191 rdf:rest N3ec354d491f748a59ba65c8ced79e8d1
    192 N89185fa9745c448d81ee4f6d083b5a0b rdf:first N750a454bbbc64a49ac9246b19c8096eb
    193 rdf:rest N57dee8906ee64bfd9d5c3419008f8b7d
    194 N8b1381bfa4e641cbaf1a614ec0f89128 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    195 schema:name Factor IX
    196 rdf:type schema:DefinedTerm
    197 N8c06f3fa746b443597919b7e9a34eb34 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    198 schema:name Middle Aged
    199 rdf:type schema:DefinedTerm
    200 N8ee92968deb743bb8e3306372dcd4285 schema:name pubmed_id
    201 schema:value 16474400
    202 rdf:type schema:PropertyValue
    203 N91f7818d671a4945bd01ab9f66fd4866 rdf:first sg:person.01237547567.14
    204 rdf:rest N130958372253465aab2cae790b98cbac
    205 N935a46d49c194800a88d4adbed0f2b07 schema:affiliation https://www.grid.ac/institutes/grid.239552.a
    206 schema:familyName Chew
    207 schema:givenName Amy
    208 rdf:type schema:Person
    209 N9579cbfd3ae146dab6cb0a46308e3cea rdf:first N4fe97376c69d4c91b0c3a0538505cfe2
    210 rdf:rest N4cf8a538e2664876b59cd8ed56f78817
    211 N95ab8606bcc84ab3ac977d6d5c9b625f rdf:first Nccd2e4a44e7d4619a982664ad19aadff
    212 rdf:rest N9628d664e568470ea89d8dd9be04e442
    213 N9628d664e568470ea89d8dd9be04e442 rdf:first N6ac3e0d435b44056b25a493a063e7f24
    214 rdf:rest N368b4b46861a4d45aed865328a9310cd
    215 N9790f97a0d454e1bac4cf132b79b0a5b rdf:first N6f64dd585fce4b69958a549d90dd23e4
    216 rdf:rest Nf1c74a7c70094fb58c251cd9d5e32b55
    217 N98ff2e3aff124947a803aa25f49ba89f schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    218 rdf:type schema:Organization
    219 N9c92479ee23d46e49b870929c1bdf733 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    220 schema:name Animals
    221 rdf:type schema:DefinedTerm
    222 N9ecf32ba32ad4031af8af9600fc5b5e0 schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    223 rdf:type schema:Organization
    224 Na5d1565ab5a848ae9e59e2c309b5ec40 schema:name Springer Nature - SN SciGraph project
    225 rdf:type schema:Organization
    226 Na756c2c92d17487d8ca4e9819dfb3d74 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    227 schema:name Hemophilia A
    228 rdf:type schema:DefinedTerm
    229 Naa2d0cc735194064aaac9e29eb8e1648 rdf:first sg:person.01104637520.44
    230 rdf:rest N1ecff20edf14416f97f37525974bd412
    231 Nb128647e60a64d0a8d43aad384babc6d rdf:first Nb339ae7e490c4f3c83613cce3a5eb8f6
    232 rdf:rest N1f9acc57c41b4a5192997b7cf1e6faeb
    233 Nb339ae7e490c4f3c83613cce3a5eb8f6 schema:affiliation https://www.grid.ac/institutes/grid.413249.9
    234 schema:familyName Rasko
    235 schema:givenName John J E
    236 rdf:type schema:Person
    237 Nb71cc538970c4037b4bf894c28aab69a schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    238 schema:name Liver
    239 rdf:type schema:DefinedTerm
    240 Nbac50b8927ae4213a9e96048d75956d0 schema:affiliation https://www.grid.ac/institutes/grid.168010.e
    241 schema:familyName Dake
    242 schema:givenName Michael
    243 rdf:type schema:Person
    244 Nbcf323d5cf4745d88886a890943c7d33 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    245 schema:name Introns
    246 rdf:type schema:DefinedTerm
    247 Nbf85230f6d55459182214cc9768f4aa3 rdf:first N73079fd8237444d1b9c018f7bfd2d6e7
    248 rdf:rest Ne93356479aa648d0bc4e1ef9edf00a69
    249 Nbfbfbd0012e1420a85dc479e77de1d18 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    250 schema:name Dogs
    251 rdf:type schema:DefinedTerm
    252 Nc09922a71af44388bca733781d289fe4 rdf:first sg:person.01035106403.91
    253 rdf:rest N6e4565e720cc459299a040dc5f0a14bc
    254 Nc2f080b9b47345d28ec84e0513afdf43 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    255 schema:name Dependovirus
    256 rdf:type schema:DefinedTerm
    257 Nc30c62cc76654e05903c42a91cd23802 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    258 schema:name Exons
    259 rdf:type schema:DefinedTerm
    260 Nc563d5a798de4dde93f88978608445e9 schema:name doi
    261 schema:value 10.1038/nm1358
    262 rdf:type schema:PropertyValue
    263 Nccd0c9d744d5485b81d5568afb9e78f3 rdf:first N5d84de8217444a06ad41a284a2511562
    264 rdf:rest N95ab8606bcc84ab3ac977d6d5c9b625f
    265 Nccd2e4a44e7d4619a982664ad19aadff schema:affiliation Ne2f2a7b554bb4a079fdcedf8e579c179
    266 schema:familyName Wright
    267 schema:givenName J Fraser
    268 rdf:type schema:Person
    269 Nd4bea0f936334169842c787535990ece schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    270 schema:name Genetic Vectors
    271 rdf:type schema:DefinedTerm
    272 Nd9596279389b42fbb1abff29429f9354 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    273 schema:name Interferon-gamma
    274 rdf:type schema:DefinedTerm
    275 Ne190f45d46eb424cb2ea3d253ad8c434 rdf:first sg:person.016335301372.89
    276 rdf:rest Nc09922a71af44388bca733781d289fe4
    277 Ne2f2a7b554bb4a079fdcedf8e579c179 schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    278 rdf:type schema:Organization
    279 Ne93356479aa648d0bc4e1ef9edf00a69 rdf:first sg:person.01200036420.15
    280 rdf:rest Naa2d0cc735194064aaac9e29eb8e1648
    281 Nec25481284f540a48f483ff396e94299 schema:affiliation https://www.grid.ac/institutes/grid.168010.e
    282 schema:familyName Razavi
    283 schema:givenName Mahmood
    284 rdf:type schema:Person
    285 Nedb2686e2c794ae58e918ce8bbabcda0 schema:affiliation N27daaf305ad043f990c74dff86777876
    286 schema:familyName Luk
    287 schema:givenName Alvin
    288 rdf:type schema:Person
    289 Nf12b8de5a1164d9e8b8a22885dcacb5e schema:affiliation https://www.grid.ac/institutes/grid.168010.e
    290 schema:familyName Nakai
    291 schema:givenName Hiroyuki
    292 rdf:type schema:Person
    293 Nf1c74a7c70094fb58c251cd9d5e32b55 rdf:first N39d86dea0de443989baeacc6b47f1c22
    294 rdf:rest N89185fa9745c448d81ee4f6d083b5a0b
    295 Nfc4e24a42cac499dbdbf088f475748d9 schema:name readcube_id
    296 schema:value 39d086bbbdc0e24974c85dee6563d2011ec42a2dbae0dd6d83e6a9414f3e28a6
    297 rdf:type schema:PropertyValue
    298 Nfd809a5e9cde4ec1ada717ae15f47606 rdf:first N02e8a29304284ce1a2f8ecbf1e3e9295
    299 rdf:rest N87f06356651c43cab9e37b15e5adeea5
    300 anzsrc-for:11 schema:inDefinedTermSet anzsrc-for:
    301 schema:name Medical and Health Sciences
    302 rdf:type schema:DefinedTerm
    303 anzsrc-for:1103 schema:inDefinedTermSet anzsrc-for:
    304 schema:name Clinical Sciences
    305 rdf:type schema:DefinedTerm
    306 sg:grant.2403943 http://pending.schema.org/fundedItem sg:pub.10.1038/nm1358
    307 rdf:type schema:MonetaryGrant
    308 sg:grant.2436938 http://pending.schema.org/fundedItem sg:pub.10.1038/nm1358
    309 rdf:type schema:MonetaryGrant
    310 sg:grant.2538929 http://pending.schema.org/fundedItem sg:pub.10.1038/nm1358
    311 rdf:type schema:MonetaryGrant
    312 sg:grant.2691684 http://pending.schema.org/fundedItem sg:pub.10.1038/nm1358
    313 rdf:type schema:MonetaryGrant
    314 sg:journal.1113716 schema:issn 1078-8956
    315 1546-170X
    316 schema:name Nature Medicine
    317 rdf:type schema:Periodical
    318 sg:person.01001004070.50 schema:affiliation https://www.grid.ac/institutes/grid.239552.a
    319 schema:familyName Sabatino
    320 schema:givenName Denise
    321 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01001004070.50
    322 rdf:type schema:Person
    323 sg:person.01035106403.91 schema:affiliation https://www.grid.ac/institutes/grid.25879.31
    324 schema:familyName Arruda
    325 schema:givenName Valder R
    326 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01035106403.91
    327 rdf:type schema:Person
    328 sg:person.01104637520.44 schema:affiliation https://www.grid.ac/institutes/grid.413575.1
    329 schema:familyName High
    330 schema:givenName Katherine A
    331 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01104637520.44
    332 rdf:type schema:Person
    333 sg:person.01145473703.03 schema:affiliation https://www.grid.ac/institutes/grid.239552.a
    334 schema:familyName Mingozzi
    335 schema:givenName Federico
    336 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01145473703.03
    337 rdf:type schema:Person
    338 sg:person.01164322460.34 schema:affiliation https://www.grid.ac/institutes/grid.168010.e
    339 schema:familyName Kay
    340 schema:givenName Mark A
    341 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01164322460.34
    342 rdf:type schema:Person
    343 sg:person.01200036420.15 schema:affiliation https://www.grid.ac/institutes/grid.251075.4
    344 schema:familyName Ertl
    345 schema:givenName Hildegund C
    346 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01200036420.15
    347 rdf:type schema:Person
    348 sg:person.01211363720.11 schema:affiliation https://www.grid.ac/institutes/grid.25879.31
    349 schema:familyName Manno
    350 schema:givenName Catherine S
    351 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01211363720.11
    352 rdf:type schema:Person
    353 sg:person.01237547567.14 schema:affiliation https://www.grid.ac/institutes/grid.25879.31
    354 schema:familyName Kaye
    355 schema:givenName Robin
    356 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01237547567.14
    357 rdf:type schema:Person
    358 sg:person.01250056754.47 schema:affiliation N27dd3d22826844d9bf762ccbdbd5958b
    359 schema:familyName Sommer
    360 schema:givenName Jürg M
    361 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01250056754.47
    362 rdf:type schema:Person
    363 sg:person.01340662554.04 schema:affiliation N20213d2581d2452cb6dd852951151c2f
    364 schema:familyName Jiang
    365 schema:givenName Haiyan
    366 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01340662554.04
    367 rdf:type schema:Person
    368 sg:person.01363703525.35 schema:affiliation https://www.grid.ac/institutes/grid.168010.e
    369 schema:familyName Glader
    370 schema:givenName Bertil
    371 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01363703525.35
    372 rdf:type schema:Person
    373 sg:person.016335301372.89 schema:affiliation N98ff2e3aff124947a803aa25f49ba89f
    374 schema:familyName Pierce
    375 schema:givenName Glenn F
    376 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.016335301372.89
    377 rdf:type schema:Person
    378 sg:person.053412353.52 schema:affiliation https://www.grid.ac/institutes/grid.414314.7
    379 schema:familyName Blatt
    380 schema:givenName Philip
    381 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.053412353.52
    382 rdf:type schema:Person
    383 sg:person.0654065663.76 schema:affiliation https://www.grid.ac/institutes/grid.411087.b
    384 schema:familyName Ozelo
    385 schema:givenName Margareth C
    386 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0654065663.76
    387 rdf:type schema:Person
    388 sg:person.0662644741.32 schema:affiliation https://www.grid.ac/institutes/grid.267308.8
    389 schema:familyName Hoots
    390 schema:givenName Keith
    391 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0662644741.32
    392 rdf:type schema:Person
    393 sg:person.0664442574.69 schema:affiliation https://www.grid.ac/institutes/grid.412689.0
    394 schema:familyName Ragni
    395 schema:givenName Margaret
    396 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0664442574.69
    397 rdf:type schema:Person
    398 sg:pub.10.1038/4743 schema:sameAs https://app.dimensions.ai/details/publication/pub.1005137439
    399 https://doi.org/10.1038/4743
    400 rdf:type schema:CreativeWork
    401 sg:pub.10.1038/4751 schema:sameAs https://app.dimensions.ai/details/publication/pub.1043560823
    402 https://doi.org/10.1038/4751
    403 rdf:type schema:CreativeWork
    404 sg:pub.10.1038/73464 schema:sameAs https://app.dimensions.ai/details/publication/pub.1023348153
    405 https://doi.org/10.1038/73464
    406 rdf:type schema:CreativeWork
    407 sg:pub.10.1038/ng0501-92 schema:sameAs https://app.dimensions.ai/details/publication/pub.1001946207
    408 https://doi.org/10.1038/ng0501-92
    409 rdf:type schema:CreativeWork
    410 sg:pub.10.1038/sj.gt.3301024 schema:sameAs https://app.dimensions.ai/details/publication/pub.1008888203
    411 https://doi.org/10.1038/sj.gt.3301024
    412 rdf:type schema:CreativeWork
    413 https://app.dimensions.ai/details/publication/pub.1083044374 schema:CreativeWork
    414 https://doi.org/10.1002/hep.1840200104 schema:sameAs https://app.dimensions.ai/details/publication/pub.1029401994
    415 rdf:type schema:CreativeWork
    416 https://doi.org/10.1002/hep.510240201 schema:sameAs https://app.dimensions.ai/details/publication/pub.1038541144
    417 rdf:type schema:CreativeWork
    418 https://doi.org/10.1006/mthe.2000.0031 schema:sameAs https://app.dimensions.ai/details/publication/pub.1045595102
    419 rdf:type schema:CreativeWork
    420 https://doi.org/10.1006/mthe.2000.0075 schema:sameAs https://app.dimensions.ai/details/publication/pub.1051938287
    421 rdf:type schema:CreativeWork
    422 https://doi.org/10.1006/mthe.2001.0491 schema:sameAs https://app.dimensions.ai/details/publication/pub.1023556799
    423 rdf:type schema:CreativeWork
    424 https://doi.org/10.1016/j.smim.2004.02.007 schema:sameAs https://app.dimensions.ai/details/publication/pub.1002351699
    425 rdf:type schema:CreativeWork
    426 https://doi.org/10.1016/j.ymthe.2005.02.021 schema:sameAs https://app.dimensions.ai/details/publication/pub.1049406390
    427 rdf:type schema:CreativeWork
    428 https://doi.org/10.1016/s0022-1759(01)00535-x schema:sameAs https://app.dimensions.ai/details/publication/pub.1010317193
    429 rdf:type schema:CreativeWork
    430 https://doi.org/10.1016/s0022-1759(03)00226-6 schema:sameAs https://app.dimensions.ai/details/publication/pub.1026782649
    431 rdf:type schema:CreativeWork
    432 https://doi.org/10.1016/s1525-0016(02)00019-9 schema:sameAs https://app.dimensions.ai/details/publication/pub.1053021243
    433 rdf:type schema:CreativeWork
    434 https://doi.org/10.1046/j.1365-2141.1998.00707.x schema:sameAs https://app.dimensions.ai/details/publication/pub.1035175583
    435 rdf:type schema:CreativeWork
    436 https://doi.org/10.1046/j.1365-2796.1997.130135000.x schema:sameAs https://app.dimensions.ai/details/publication/pub.1036948431
    437 rdf:type schema:CreativeWork
    438 https://doi.org/10.1074/jbc.270.10.5276 schema:sameAs https://app.dimensions.ai/details/publication/pub.1015848421
    439 rdf:type schema:CreativeWork
    440 https://doi.org/10.1089/104303404322886138 schema:sameAs https://app.dimensions.ai/details/publication/pub.1059202704
    441 rdf:type schema:CreativeWork
    442 https://doi.org/10.1128/jvi.78.24.13678-13686.2004 schema:sameAs https://app.dimensions.ai/details/publication/pub.1011327932
    443 rdf:type schema:CreativeWork
    444 https://doi.org/10.1128/jvi.78.6.3110-3122.2004 schema:sameAs https://app.dimensions.ai/details/publication/pub.1013436925
    445 rdf:type schema:CreativeWork
    446 https://doi.org/10.1182/blood-2002-02-0589 schema:sameAs https://app.dimensions.ai/details/publication/pub.1036180752
    447 rdf:type schema:CreativeWork
    448 https://doi.org/10.1182/blood-2002-10-3296 schema:sameAs https://app.dimensions.ai/details/publication/pub.1003619350
    449 rdf:type schema:CreativeWork
    450 https://doi.org/10.1182/blood-2003-01-0292 schema:sameAs https://app.dimensions.ai/details/publication/pub.1021429034
    451 rdf:type schema:CreativeWork
    452 https://doi.org/10.1182/blood-2004-07-2908 schema:sameAs https://app.dimensions.ai/details/publication/pub.1026834251
    453 rdf:type schema:CreativeWork
    454 https://doi.org/10.1182/blood-2004-10-3867 schema:sameAs https://app.dimensions.ai/details/publication/pub.1018702261
    455 rdf:type schema:CreativeWork
    456 https://doi.org/10.1182/blood-2005-10-4035 schema:sameAs https://app.dimensions.ai/details/publication/pub.1035241251
    457 rdf:type schema:CreativeWork
    458 https://doi.org/10.1182/blood.v99.8.2670 schema:sameAs https://app.dimensions.ai/details/publication/pub.1019564621
    459 rdf:type schema:CreativeWork
    460 https://www.grid.ac/institutes/grid.168010.e schema:alternateName Stanford University
    461 schema:name Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, California 94305-5208, USA.
    462 rdf:type schema:Organization
    463 https://www.grid.ac/institutes/grid.239552.a schema:alternateName Children's Hospital of Philadelphia
    464 schema:name Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA.
    465 The Children's Hospital of Philadelphia, and
    466 rdf:type schema:Organization
    467 https://www.grid.ac/institutes/grid.251075.4 schema:alternateName Wistar Institute
    468 schema:name The Children's Hospital of Philadelphia, and
    469 Wistar Institute, 3601 Spruce Street, Philadelphia, Pennsylvania 19104, USA.
    470 rdf:type schema:Organization
    471 https://www.grid.ac/institutes/grid.25879.31 schema:alternateName University of Pennsylvania
    472 schema:name The Children's Hospital of Philadelphia, and
    473 University of Pennsylvania School of Medicine Presbyterian Medical Center, MAB, Suite 103, 39th & Market Street, Philadelphia, Pennsylvania 19104, USA.
    474 University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA.
    475 rdf:type schema:Organization
    476 https://www.grid.ac/institutes/grid.267308.8 schema:alternateName The University of Texas Health Science Center at Houston
    477 schema:name University of Texas Houston Health Science Center, 1515 Holcombe Boulevard, Houston, Texas 77030, USA.
    478 rdf:type schema:Organization
    479 https://www.grid.ac/institutes/grid.411087.b schema:alternateName State University of Campinas
    480 schema:name University of Campinas, State University of Campinas, Caixa Postal 6198, CEP, 13083-970, Campinas-Sao Paolo, Brazil.
    481 rdf:type schema:Organization
    482 https://www.grid.ac/institutes/grid.412689.0 schema:alternateName University of Pittsburgh Medical Center
    483 schema:name University of Pittsburgh Medical Center, Hemophilia Center of Western Pennsylvania, 3636 Boulevard of the Allies, Pittsburgh, Pennsylvania 15213, USA.
    484 rdf:type schema:Organization
    485 https://www.grid.ac/institutes/grid.413249.9 schema:alternateName Royal Prince Alfred Hospital
    486 schema:name Royal Prince Alfred Hospital, Centenary Institute of Cancer Medicine & Cell Biology, Locked Bag No 6, Newtown NSW 2042, Australia.
    487 rdf:type schema:Organization
    488 https://www.grid.ac/institutes/grid.413575.1 schema:alternateName Howard Hughes Medical Institute
    489 schema:name Howard Hughes Medical Institute, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA.
    490 The Children's Hospital of Philadelphia, and
    491 University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA.
    492 rdf:type schema:Organization
    493 https://www.grid.ac/institutes/grid.414314.7 schema:alternateName Christiana Hospital
    494 schema:name Christiana Care, Christiana Hospital, 4755 Ogletown-Stanton Road, Newark, Delaware 19713, USA.
    495 rdf:type schema:Organization
    496 https://www.grid.ac/institutes/grid.5386.8 schema:alternateName Cornell University
    497 schema:name University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA.
    498 Weill Medical College of Cornell University, 525 East 68th Street, New York, New York 10012, USA.
    499 rdf:type schema:Organization
     




    Preview window. Press ESC to close (or click here)


    ...