Promoterless gene targeting without nucleases ameliorates haemophilia B in mice View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2014-10-29

AUTHORS

A. Barzel, N. K. Paulk, Y. Shi, Y. Huang, K. Chu, F. Zhang, P. N. Valdmanis, L. P. Spector, M. H. Porteus, K. M. Gaensler, M. A. Kay

ABSTRACT

Promoterless recombinant adeno-associated virus is used without nucleases to target the human coagulation factor IX gene to the liver-expressed albumin locus in haemophilia B mice, with an on-target integration into ∼0.5% of the albumin alleles in hepatocytes; stable F9 plasma levels at 7–20% of normal were obtained, leading to normal coagulation times in treated factor-IX-deficient mice. More... »

PAGES

360-364

Journal

TITLE

Nature

ISSUE

7534

VOLUME

517

Related Patents

  • Tolerogenic Synthetic Nanocarriers To Reduce Or Prevent Anaphylaxis In Response To A Non-Allergenic Antigen
  • Tolerogenic Synthetic Nanocarriers For Generating Cd8+ Regulatory T Cells
  • Method For Providing Polymeric Synthetic Nanocarriers For Generating Antigen-Specific Tolerance Immune Responses
  • Methods Related To Administering Immunosuppressants And Therapeutic Macromolecules At A Reduced Pharmacodynamically Effective Dose
  • Methods And Compositions For Attenuating Exon Skipping Anti-Viral Transfer Vector Immune Responses
  • Methods Of Administering Immunosuppressants Having A Specified Pharmacodynamic Effective Life And Therapeutic Macromolecules For The Induction Of Immune Tolerance
  • Methods For Providing Polymeric Synthetic Nanocarriers For Generating Antigen-Specific Tolerance Immune Responses
  • Tolerogenic Synthetic Nanocarriers For Inducing Regulatory B Cells
  • Genome Editing Methods And Constructs
  • Methods Providing A Therapeutic Macromolecule And Synthetic Nanocarriers Comprising Immunosuppressant Locally And Concomitantly To Reduce Both Type I And Type Iv Hypersensitivity
  • Methods Comprising Dosing Combinations For Reducing Undesired Humoral Immune Responses
  • Methods And Compositions For Enhancing Targeted Transgene Integration
  • Monitoring Gene Therapy
  • Biodegradable Amino-Ester Nanomaterials For Nucleic Acid Delivery
  • Compositions And Methods For Internalizing Enzymes
  • Nucleic Acid Constructs And Methods Of Use
  • Genome Editing Without Nucleases
  • Methods And Compositions For Attenuating Gene Expression Modulating Anti-Viral Transfer Vector Immune Responses
  • Methods And Compositions Related To Combined Treatment With Antiinflammatories And Synthetic Nanocarriers Comprising An Immunosuppressant
  • Tolerogenic Synthetic Nanocarriers For Antigen-Specific Deletion Of T Effector Cells
  • Adeno-Associated Virus Compositions For Restoring F8 Gene Function And Methods Of Use Thereof
  • Tolerogenic Synthetic Nanocarriers For Antigen-Specific Deletion Of T Effector Cells
  • Benzene-1,3,5-Tricarboxamide Derivatives And Uses Thereof
  • Methods Providing A Therapeutic Macromolecule And Synthetic Nanocarriers Comprising Immunosuppressant Locally And Concomitantly To Reduce Both Type I And Type Iv Hypersensitivity
  • Nucleic Acid Molecules Containing Spacers And Methods Of Use Thereof
  • Genome Editing Without Nucleases
  • Tolerogenic Synthetic Nanocarriers For Generating Cd8+ Regulatory T Cells
  • Novel Recombinant Adeno-Associated Virus Capsids Resistant To Pre-Existing Human Neutralizing Antibodies
  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/nature13864

    DOI

    http://dx.doi.org/10.1038/nature13864

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1039253377

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/25363772


    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/2008/11", 
            "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
            "name": "Medical and Health Sciences", 
            "type": "DefinedTerm"
          }, 
          {
            "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"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Alleles", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Animals", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Codon, Terminator", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Dependovirus", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Disease Models, Animal", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Endonucleases", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Factor IX", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Female", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Gene Targeting", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Hemophilia B", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Hepatocytes", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Humans", 
            "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": "Mice, Inbred C57BL", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Promoter Regions, Genetic", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Ribosomes", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Serum Albumin", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Transgenes", 
            "type": "DefinedTerm"
          }
        ], 
        "author": [
          {
            "affiliation": {
              "alternateName": "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Barzel", 
            "givenName": "A.", 
            "id": "sg:person.0642325355.35", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0642325355.35"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Paulk", 
            "givenName": "N. K.", 
            "id": "sg:person.01030377225.21", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01030377225.21"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Department of Medicine, Box 1270, UCSF, San Francisco, California 94143-1270, USA", 
              "id": "http://www.grid.ac/institutes/grid.266102.1", 
              "name": [
                "Department of Medicine, Box 1270, UCSF, San Francisco, California 94143-1270, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Shi", 
            "givenName": "Y.", 
            "id": "sg:person.01255554316.39", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01255554316.39"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Huang", 
            "givenName": "Y.", 
            "id": "sg:person.0606725436.68", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0606725436.68"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Chu", 
            "givenName": "K.", 
            "id": "sg:person.01136776010.50", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01136776010.50"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Zhang", 
            "givenName": "F.", 
            "id": "sg:person.0670052524.50", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0670052524.50"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Valdmanis", 
            "givenName": "P. N.", 
            "id": "sg:person.0657215642.18", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0657215642.18"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Spector", 
            "givenName": "L. P.", 
            "id": "sg:person.0756230213.17", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0756230213.17"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Department of Pediatrics, 269 Campus Drive, Lorry Lokey Stem Cell Research Building, Room G3045, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Department of Pediatrics, 269 Campus Drive, Lorry Lokey Stem Cell Research Building, Room G3045, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Porteus", 
            "givenName": "M. H.", 
            "id": "sg:person.01115066120.22", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01115066120.22"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Department of Medicine, Box 1270, UCSF, San Francisco, California 94143-1270, USA", 
              "id": "http://www.grid.ac/institutes/grid.266102.1", 
              "name": [
                "Department of Medicine, Box 1270, UCSF, San Francisco, California 94143-1270, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Gaensler", 
            "givenName": "K. M.", 
            "id": "sg:person.013335621115.52", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.013335621115.52"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA", 
              "id": "http://www.grid.ac/institutes/None", 
              "name": [
                "Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Kay", 
            "givenName": "M. 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/nature10177", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1041862601", 
              "https://doi.org/10.1038/nature10177"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/nature10660", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1008035308", 
              "https://doi.org/10.1038/nature10660"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/nprot.2008.80", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1026086634", 
              "https://doi.org/10.1038/nprot.2008.80"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/nature12875", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1007748656", 
              "https://doi.org/10.1038/nature12875"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/gt.2013.22", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1038267497", 
              "https://doi.org/10.1038/gt.2013.22"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/nature04791", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1047286924", 
              "https://doi.org/10.1038/nature04791"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/nbt.2623", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1009511624", 
              "https://doi.org/10.1038/nbt.2623"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/nbt1231", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1018588042", 
              "https://doi.org/10.1038/nbt1231"
            ], 
            "type": "CreativeWork"
          }
        ], 
        "datePublished": "2014-10-29", 
        "datePublishedReg": "2014-10-29", 
        "description": "Promoterless recombinant adeno-associated virus is used without nucleases to target the human coagulation factor IX gene to the liver-expressed albumin locus in haemophilia B mice, with an on-target integration into \u223c0.5% of the albumin alleles in hepatocytes; stable F9 plasma levels at 7\u201320% of normal were obtained, leading to normal coagulation times in treated factor-IX-deficient mice.", 
        "genre": "article", 
        "id": "sg:pub.10.1038/nature13864", 
        "isAccessibleForFree": true, 
        "isFundedItemOf": [
          {
            "id": "sg:grant.2684533", 
            "type": "MonetaryGrant"
          }, 
          {
            "id": "sg:grant.2705250", 
            "type": "MonetaryGrant"
          }, 
          {
            "id": "sg:grant.3623894", 
            "type": "MonetaryGrant"
          }, 
          {
            "id": "sg:grant.2537300", 
            "type": "MonetaryGrant"
          }
        ], 
        "isPartOf": [
          {
            "id": "sg:journal.1018957", 
            "issn": [
              "0028-0836", 
              "1476-4687"
            ], 
            "name": "Nature", 
            "publisher": "Springer Nature", 
            "type": "Periodical"
          }, 
          {
            "issueNumber": "7534", 
            "type": "PublicationIssue"
          }, 
          {
            "type": "PublicationVolume", 
            "volumeNumber": "517"
          }
        ], 
        "keywords": [
          "normal coagulation times", 
          "hemophilia B mice", 
          "B mice", 
          "plasma levels", 
          "deficient mice", 
          "adeno-associated virus", 
          "recombinant adeno-associated virus", 
          "human coagulation factor IX gene", 
          "hemophilia B", 
          "mice", 
          "coagulation time", 
          "coagulation factor IX gene", 
          "factor IX gene", 
          "IX gene", 
          "virus", 
          "hepatocytes", 
          "genes", 
          "albumin locus", 
          "alleles", 
          "levels", 
          "time", 
          "loci", 
          "nuclease", 
          "promoterless gene", 
          "integration", 
          "target integration"
        ], 
        "name": "Promoterless gene targeting without nucleases ameliorates haemophilia B in mice", 
        "pagination": "360-364", 
        "productId": [
          {
            "name": "dimensions_id", 
            "type": "PropertyValue", 
            "value": [
              "pub.1039253377"
            ]
          }, 
          {
            "name": "doi", 
            "type": "PropertyValue", 
            "value": [
              "10.1038/nature13864"
            ]
          }, 
          {
            "name": "pubmed_id", 
            "type": "PropertyValue", 
            "value": [
              "25363772"
            ]
          }
        ], 
        "sameAs": [
          "https://doi.org/10.1038/nature13864", 
          "https://app.dimensions.ai/details/publication/pub.1039253377"
        ], 
        "sdDataset": "articles", 
        "sdDatePublished": "2022-10-01T06:39", 
        "sdLicense": "https://scigraph.springernature.com/explorer/license/", 
        "sdPublisher": {
          "name": "Springer Nature - SN SciGraph project", 
          "type": "Organization"
        }, 
        "sdSource": "s3://com-springernature-scigraph/baseset/20221001/entities/gbq_results/article/article_639.jsonl", 
        "type": "ScholarlyArticle", 
        "url": "https://doi.org/10.1038/nature13864"
      }
    ]
     

    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/nature13864'

    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/nature13864'

    Turtle is a human-readable linked data format.

    curl -H 'Accept: text/turtle' 'https://scigraph.springernature.com/pub.10.1038/nature13864'

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

    curl -H 'Accept: application/rdf+xml' 'https://scigraph.springernature.com/pub.10.1038/nature13864'


     

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

    282 TRIPLES      21 PREDICATES      79 URIs      63 LITERALS      27 BLANK NODES

    Subject Predicate Object
    1 sg:pub.10.1038/nature13864 schema:about N075497fc878546f286732aaa8bde51eb
    2 N0a701a7b72384e75a7e53ba64b30a7f1
    3 N2137427bf20a4bc683e7e1068d0f8104
    4 N2b4243595df7460fb1e09a640f9ea515
    5 N31073cceb4ac4dcfbc254a462dceed3e
    6 N51309c12004549309bd12d93edd25d7c
    7 N6274552f62284ec088c68d19505c6fcd
    8 N7011f55c493e424ebf187d5a35a4c769
    9 N86a188745f834ebfa238b4ad76202c97
    10 N90944fc3083649fd8a462c247dcafd56
    11 Na02cad72995c41779c0a1b319b31b94a
    12 Naf3876b3563d4698b708705491f2a58b
    13 Nb65bcd93196a49558a51459037f87048
    14 Nc321b7b56c504ca3a3977be269271c57
    15 Nd71ac932f43640daa52da5dcd2df805e
    16 Nd82292addc824d9986cd6fb7c34a54c5
    17 Nd82e4689f0fb4b2583e83ba8da21067f
    18 Ndbd2b2fd8cfb42708373643a7cb1da60
    19 Neb36d1050dcc469a84be3baf5f646db3
    20 Nf73050aa30604dcaaf5016df8137a6a1
    21 anzsrc-for:11
    22 anzsrc-for:1103
    23 schema:author N17b2ed5d771f430e8379e33ccdd32336
    24 schema:citation sg:pub.10.1038/gt.2013.22
    25 sg:pub.10.1038/nature04791
    26 sg:pub.10.1038/nature10177
    27 sg:pub.10.1038/nature10660
    28 sg:pub.10.1038/nature12875
    29 sg:pub.10.1038/nbt.2623
    30 sg:pub.10.1038/nbt1231
    31 sg:pub.10.1038/nprot.2008.80
    32 schema:datePublished 2014-10-29
    33 schema:datePublishedReg 2014-10-29
    34 schema:description Promoterless recombinant adeno-associated virus is used without nucleases to target the human coagulation factor IX gene to the liver-expressed albumin locus in haemophilia B mice, with an on-target integration into ∼0.5% of the albumin alleles in hepatocytes; stable F9 plasma levels at 7–20% of normal were obtained, leading to normal coagulation times in treated factor-IX-deficient mice.
    35 schema:genre article
    36 schema:isAccessibleForFree true
    37 schema:isPartOf Ncdce881f733b4a6b92d61247d29e8adf
    38 Nd019f2eab561422dbdc6301626cb8ca0
    39 sg:journal.1018957
    40 schema:keywords B mice
    41 IX gene
    42 adeno-associated virus
    43 albumin locus
    44 alleles
    45 coagulation factor IX gene
    46 coagulation time
    47 deficient mice
    48 factor IX gene
    49 genes
    50 hemophilia B
    51 hemophilia B mice
    52 hepatocytes
    53 human coagulation factor IX gene
    54 integration
    55 levels
    56 loci
    57 mice
    58 normal coagulation times
    59 nuclease
    60 plasma levels
    61 promoterless gene
    62 recombinant adeno-associated virus
    63 target integration
    64 time
    65 virus
    66 schema:name Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
    67 schema:pagination 360-364
    68 schema:productId N334ef862b21545129c962224899156c4
    69 N8ae361bbd13c419f8ea7a99b0b2de871
    70 Nd41760fca7be48bfb37b4fde9099cea0
    71 schema:sameAs https://app.dimensions.ai/details/publication/pub.1039253377
    72 https://doi.org/10.1038/nature13864
    73 schema:sdDatePublished 2022-10-01T06:39
    74 schema:sdLicense https://scigraph.springernature.com/explorer/license/
    75 schema:sdPublisher Nb07f6ddeeb5a4f0f80775077e70ce83b
    76 schema:url https://doi.org/10.1038/nature13864
    77 sgo:license sg:explorer/license/
    78 sgo:sdDataset articles
    79 rdf:type schema:ScholarlyArticle
    80 N075497fc878546f286732aaa8bde51eb schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    81 schema:name Animals
    82 rdf:type schema:DefinedTerm
    83 N0a701a7b72384e75a7e53ba64b30a7f1 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    84 schema:name Gene Targeting
    85 rdf:type schema:DefinedTerm
    86 N10956230da314027aee1d65a6d94788a rdf:first sg:person.0606725436.68
    87 rdf:rest Nbd2951b736a043ed8bd636e3d3d7544f
    88 N17b2ed5d771f430e8379e33ccdd32336 rdf:first sg:person.0642325355.35
    89 rdf:rest N67a71fb98fd940da9d137b509f1523dd
    90 N2137427bf20a4bc683e7e1068d0f8104 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    91 schema:name Mice
    92 rdf:type schema:DefinedTerm
    93 N2b4243595df7460fb1e09a640f9ea515 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    94 schema:name Male
    95 rdf:type schema:DefinedTerm
    96 N31073cceb4ac4dcfbc254a462dceed3e schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    97 schema:name Disease Models, Animal
    98 rdf:type schema:DefinedTerm
    99 N334ef862b21545129c962224899156c4 schema:name doi
    100 schema:value 10.1038/nature13864
    101 rdf:type schema:PropertyValue
    102 N51309c12004549309bd12d93edd25d7c schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    103 schema:name Alleles
    104 rdf:type schema:DefinedTerm
    105 N56c7b04cc3f04253863e626f441dc0b2 rdf:first sg:person.0670052524.50
    106 rdf:rest Na951ee35c8df4e70bc9ef0c336a1ac2e
    107 N6274552f62284ec088c68d19505c6fcd schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    108 schema:name Hemophilia B
    109 rdf:type schema:DefinedTerm
    110 N67a71fb98fd940da9d137b509f1523dd rdf:first sg:person.01030377225.21
    111 rdf:rest N98553317167c424f834b0c54f1b462ab
    112 N7011f55c493e424ebf187d5a35a4c769 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    113 schema:name Hepatocytes
    114 rdf:type schema:DefinedTerm
    115 N8322b42a4f284cd096c79a852f3cc129 rdf:first sg:person.01115066120.22
    116 rdf:rest Ne265049fb061461f961fb3c02dec877d
    117 N86a188745f834ebfa238b4ad76202c97 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    118 schema:name Serum Albumin
    119 rdf:type schema:DefinedTerm
    120 N8ae361bbd13c419f8ea7a99b0b2de871 schema:name pubmed_id
    121 schema:value 25363772
    122 rdf:type schema:PropertyValue
    123 N90944fc3083649fd8a462c247dcafd56 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    124 schema:name Codon, Terminator
    125 rdf:type schema:DefinedTerm
    126 N98553317167c424f834b0c54f1b462ab rdf:first sg:person.01255554316.39
    127 rdf:rest N10956230da314027aee1d65a6d94788a
    128 Na02cad72995c41779c0a1b319b31b94a schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    129 schema:name Endonucleases
    130 rdf:type schema:DefinedTerm
    131 Na951ee35c8df4e70bc9ef0c336a1ac2e rdf:first sg:person.0657215642.18
    132 rdf:rest Nec40bec853fd4d64bdd20e672775feaf
    133 Naf3876b3563d4698b708705491f2a58b schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    134 schema:name Dependovirus
    135 rdf:type schema:DefinedTerm
    136 Nb07f6ddeeb5a4f0f80775077e70ce83b schema:name Springer Nature - SN SciGraph project
    137 rdf:type schema:Organization
    138 Nb65bcd93196a49558a51459037f87048 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    139 schema:name Female
    140 rdf:type schema:DefinedTerm
    141 Nbd2951b736a043ed8bd636e3d3d7544f rdf:first sg:person.01136776010.50
    142 rdf:rest N56c7b04cc3f04253863e626f441dc0b2
    143 Nc321b7b56c504ca3a3977be269271c57 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    144 schema:name Mice, Inbred C57BL
    145 rdf:type schema:DefinedTerm
    146 Ncdce881f733b4a6b92d61247d29e8adf schema:issueNumber 7534
    147 rdf:type schema:PublicationIssue
    148 Nd019f2eab561422dbdc6301626cb8ca0 schema:volumeNumber 517
    149 rdf:type schema:PublicationVolume
    150 Nd077fb82deb4486ba220a18e384c1709 rdf:first sg:person.01164322460.34
    151 rdf:rest rdf:nil
    152 Nd41760fca7be48bfb37b4fde9099cea0 schema:name dimensions_id
    153 schema:value pub.1039253377
    154 rdf:type schema:PropertyValue
    155 Nd71ac932f43640daa52da5dcd2df805e schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    156 schema:name Transgenes
    157 rdf:type schema:DefinedTerm
    158 Nd82292addc824d9986cd6fb7c34a54c5 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    159 schema:name Ribosomes
    160 rdf:type schema:DefinedTerm
    161 Nd82e4689f0fb4b2583e83ba8da21067f schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    162 schema:name Humans
    163 rdf:type schema:DefinedTerm
    164 Ndbd2b2fd8cfb42708373643a7cb1da60 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    165 schema:name Factor IX
    166 rdf:type schema:DefinedTerm
    167 Ne265049fb061461f961fb3c02dec877d rdf:first sg:person.013335621115.52
    168 rdf:rest Nd077fb82deb4486ba220a18e384c1709
    169 Neb36d1050dcc469a84be3baf5f646db3 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    170 schema:name Promoter Regions, Genetic
    171 rdf:type schema:DefinedTerm
    172 Nec40bec853fd4d64bdd20e672775feaf rdf:first sg:person.0756230213.17
    173 rdf:rest N8322b42a4f284cd096c79a852f3cc129
    174 Nf73050aa30604dcaaf5016df8137a6a1 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    175 schema:name Liver
    176 rdf:type schema:DefinedTerm
    177 anzsrc-for:11 schema:inDefinedTermSet anzsrc-for:
    178 schema:name Medical and Health Sciences
    179 rdf:type schema:DefinedTerm
    180 anzsrc-for:1103 schema:inDefinedTermSet anzsrc-for:
    181 schema:name Clinical Sciences
    182 rdf:type schema:DefinedTerm
    183 sg:grant.2537300 http://pending.schema.org/fundedItem sg:pub.10.1038/nature13864
    184 rdf:type schema:MonetaryGrant
    185 sg:grant.2684533 http://pending.schema.org/fundedItem sg:pub.10.1038/nature13864
    186 rdf:type schema:MonetaryGrant
    187 sg:grant.2705250 http://pending.schema.org/fundedItem sg:pub.10.1038/nature13864
    188 rdf:type schema:MonetaryGrant
    189 sg:grant.3623894 http://pending.schema.org/fundedItem sg:pub.10.1038/nature13864
    190 rdf:type schema:MonetaryGrant
    191 sg:journal.1018957 schema:issn 0028-0836
    192 1476-4687
    193 schema:name Nature
    194 schema:publisher Springer Nature
    195 rdf:type schema:Periodical
    196 sg:person.01030377225.21 schema:affiliation grid-institutes:None
    197 schema:familyName Paulk
    198 schema:givenName N. K.
    199 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01030377225.21
    200 rdf:type schema:Person
    201 sg:person.01115066120.22 schema:affiliation grid-institutes:None
    202 schema:familyName Porteus
    203 schema:givenName M. H.
    204 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01115066120.22
    205 rdf:type schema:Person
    206 sg:person.01136776010.50 schema:affiliation grid-institutes:None
    207 schema:familyName Chu
    208 schema:givenName K.
    209 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01136776010.50
    210 rdf:type schema:Person
    211 sg:person.01164322460.34 schema:affiliation grid-institutes:None
    212 schema:familyName Kay
    213 schema:givenName M. A.
    214 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01164322460.34
    215 rdf:type schema:Person
    216 sg:person.01255554316.39 schema:affiliation grid-institutes:grid.266102.1
    217 schema:familyName Shi
    218 schema:givenName Y.
    219 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01255554316.39
    220 rdf:type schema:Person
    221 sg:person.013335621115.52 schema:affiliation grid-institutes:grid.266102.1
    222 schema:familyName Gaensler
    223 schema:givenName K. M.
    224 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.013335621115.52
    225 rdf:type schema:Person
    226 sg:person.0606725436.68 schema:affiliation grid-institutes:None
    227 schema:familyName Huang
    228 schema:givenName Y.
    229 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0606725436.68
    230 rdf:type schema:Person
    231 sg:person.0642325355.35 schema:affiliation grid-institutes:None
    232 schema:familyName Barzel
    233 schema:givenName A.
    234 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0642325355.35
    235 rdf:type schema:Person
    236 sg:person.0657215642.18 schema:affiliation grid-institutes:None
    237 schema:familyName Valdmanis
    238 schema:givenName P. N.
    239 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0657215642.18
    240 rdf:type schema:Person
    241 sg:person.0670052524.50 schema:affiliation grid-institutes:None
    242 schema:familyName Zhang
    243 schema:givenName F.
    244 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0670052524.50
    245 rdf:type schema:Person
    246 sg:person.0756230213.17 schema:affiliation grid-institutes:None
    247 schema:familyName Spector
    248 schema:givenName L. P.
    249 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0756230213.17
    250 rdf:type schema:Person
    251 sg:pub.10.1038/gt.2013.22 schema:sameAs https://app.dimensions.ai/details/publication/pub.1038267497
    252 https://doi.org/10.1038/gt.2013.22
    253 rdf:type schema:CreativeWork
    254 sg:pub.10.1038/nature04791 schema:sameAs https://app.dimensions.ai/details/publication/pub.1047286924
    255 https://doi.org/10.1038/nature04791
    256 rdf:type schema:CreativeWork
    257 sg:pub.10.1038/nature10177 schema:sameAs https://app.dimensions.ai/details/publication/pub.1041862601
    258 https://doi.org/10.1038/nature10177
    259 rdf:type schema:CreativeWork
    260 sg:pub.10.1038/nature10660 schema:sameAs https://app.dimensions.ai/details/publication/pub.1008035308
    261 https://doi.org/10.1038/nature10660
    262 rdf:type schema:CreativeWork
    263 sg:pub.10.1038/nature12875 schema:sameAs https://app.dimensions.ai/details/publication/pub.1007748656
    264 https://doi.org/10.1038/nature12875
    265 rdf:type schema:CreativeWork
    266 sg:pub.10.1038/nbt.2623 schema:sameAs https://app.dimensions.ai/details/publication/pub.1009511624
    267 https://doi.org/10.1038/nbt.2623
    268 rdf:type schema:CreativeWork
    269 sg:pub.10.1038/nbt1231 schema:sameAs https://app.dimensions.ai/details/publication/pub.1018588042
    270 https://doi.org/10.1038/nbt1231
    271 rdf:type schema:CreativeWork
    272 sg:pub.10.1038/nprot.2008.80 schema:sameAs https://app.dimensions.ai/details/publication/pub.1026086634
    273 https://doi.org/10.1038/nprot.2008.80
    274 rdf:type schema:CreativeWork
    275 grid-institutes:None schema:alternateName Department of Pediatrics, 269 Campus Drive, Lorry Lokey Stem Cell Research Building, Room G3045, Stanford, California 94305-5164, USA
    276 Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
    277 schema:name Department of Pediatrics, 269 Campus Drive, Lorry Lokey Stem Cell Research Building, Room G3045, Stanford, California 94305-5164, USA
    278 Departments of Pediatrics and Genetics, 269 Campus Drive, CCSR Building, Room 2105, Stanford, California 94305-5164, USA
    279 rdf:type schema:Organization
    280 grid-institutes:grid.266102.1 schema:alternateName Department of Medicine, Box 1270, UCSF, San Francisco, California 94143-1270, USA
    281 schema:name Department of Medicine, Box 1270, UCSF, San Francisco, California 94143-1270, USA
    282 rdf:type schema:Organization
     




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


    ...