Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

1992-04

AUTHORS

E Gussoni, G K Pavlath, A M Lanctot, K R Sharma, R G Miller, L Steinman, H M Blau

ABSTRACT

Gene delivery by transplantation of normal myoblasts has been proposed as a treatment of the primary defect, lack of the muscle protein dystrophin, that causes Duchenne muscular dystrophy (DMD), a lethal human muscle degenerative disorder. To test this possibility, we transplanted normal myoblasts from a father or an unaffected sibling into the muscle of eight boys with DMD, and assessed their production of dystrophin. Three patients with deletions in the dystrophin gene expressed normal dystrophin transcripts in muscle biopsy specimens taken from the transplant site one month after myoblast injection. Using the polymerase chain reaction we established that the dystrophin in these biopsies derived from donor myoblast DNA. These results show that transplanted myoblasts persist and produce dystrophin in muscle fibres of DMD patients. More... »

PAGES

435-438

Journal

TITLE

Nature

ISSUE

6368

VOLUME

356

Author Affiliations

Related Patents

  • Methods And Compositions For The Repair And/Or Regeneration Of Damaged Myocardium
  • Methods And Compositions For The Repair And/Or Regeneration Of Damaged Myocardium Using Cytokines And Variants Thereof
  • Methods And Compositions For The Repair And/Or Regeneration Of Damaged Myocardium Using Cytokines And Variants Thereof
  • Myocardial Grafts And Cellular Compositions Useful For Same
  • Transplants For Myocardial Scars
  • Bone Augmentation Utilizing Muscle-Derived Progenitor Compositions In Biocompatible Matrix, And Treatments Thereof
  • Encapsulated Cell Indicator System
  • Automated Cell Processor
  • Transcription Control Element For Increasing Gene Expression In Myoblasts
  • Rapid Preparation Of Stem Cell Matrices For Use In Tissue And Organ Treatment And Repair
  • Soft Tissue And Bone Augmentation And Bulking Utilizing Muscle-Derived Progenitor Cells, Compositions And Treatments Thereof
  • Myocardial Grafts And Cellular Compositions
  • Methods And Compositions For The Repair And/Or Regeneration Of Damaged Myocardium
  • Muscle-Derived Cells (Mdcs) For Promoting And Enhancing Nerve Repair And Regeneration
  • Nucleic Acid Constructs And Cells, And Methods Utilizing Same For Modifying The Electrophysiological Function Of Excitable Tissues
  • Muscle-Derived Cells (Mdcs) For Treating Muscle- Or Bone-Related Injury Or Dysfunction
  • Nucleic Acid Constructs And Cells, And Methods Utilizing Same For Modifying The Electrophysiological Function Of Excitable Tissues
  • Myocardial Grafts And Cellular Compositions
  • Muscle-Derived Cells (Mdcs) For Treating Muscle-Or Bone-Related Injury Or Dysfunction
  • Muscle-Derived Cells (Mdcs) For Augmenting Or Bulking Urethral Sphincter-Muscle Tissue
  • Arterial Introduction Of Myoblasts
  • Methods And Compositions For The Repair And/Or Regeneration Of Damaged Myocardium
  • Methods And Compositions For The Repair And/Or Regeneration Of Damaged Myocardium
  • Muscle Derived Cells For The Treatment Of Urinary Tract Pathologies And Methods Of Making And Using The Same
  • Methods And Compositions For The Repair And/Or Regeneration Of Damaged Myocardium
  • Soft Tissue And Bone Augmentation And Bulking Utilizing Muscle-Derived Progenito Compositions, And Treatments Thereof
  • Nucleic Acid Constructs And Cells, And Methods Utilizing Same For Modifying The Electrophysiological Function Of Excitable Tissues
  • Methods And Compositions For The Repair And/Or Regeneration Of Damaged Myocardium
  • Myoblast Therapy For Cosmetic Treatment
  • Soft Tissue And Bone Augmentation And Bulking Utilizing Muscle-Derived Progenitor Cells, Compositions And Treatments Thereof
  • Non-Human Mammal Having A Graft And Methods Of Delivering Protein To Myocardial Tissue
  • Myocardial Grafts And Cellular Compositions
  • Muscle Derived Cells For The Treatment Of Urinary Tract Pathologies And Methods Of Making And Using Same
  • Muscle-Derived Cells (Mdcs) For Treating Muscle- Or Bone-Related Injury Or Dysfunction
  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/356435a0

    DOI

    http://dx.doi.org/10.1038/356435a0

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1025801133

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/1557125


    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/2008/1004", 
            "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
            "name": "Medical Biotechnology", 
            "type": "DefinedTerm"
          }, 
          {
            "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/10", 
            "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
            "name": "Technology", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Adult", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Base Sequence", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Child", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Dystrophin", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Exons", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "HLA Antigens", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Histocompatibility Testing", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Humans", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Molecular Sequence Data", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Muscles", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Muscular Dystrophies", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Oligodeoxyribonucleotides", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Oligonucleotide Probes", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Polymerase Chain Reaction", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "RNA, Messenger", 
            "type": "DefinedTerm"
          }, 
          {
            "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
            "name": "Transcription, Genetic", 
            "type": "DefinedTerm"
          }
        ], 
        "author": [
          {
            "affiliation": {
              "alternateName": "Stanford University", 
              "id": "https://www.grid.ac/institutes/grid.168010.e", 
              "name": [
                "Department of Neurology and Neurological Sciences, Stanford University School of Medicine, California 94305."
              ], 
              "type": "Organization"
            }, 
            "familyName": "Gussoni", 
            "givenName": "E", 
            "id": "sg:person.0605476717.51", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0605476717.51"
            ], 
            "type": "Person"
          }, 
          {
            "familyName": "Pavlath", 
            "givenName": "G K", 
            "id": "sg:person.01223601511.47", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01223601511.47"
            ], 
            "type": "Person"
          }, 
          {
            "familyName": "Lanctot", 
            "givenName": "A M", 
            "id": "sg:person.01150213550.00", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01150213550.00"
            ], 
            "type": "Person"
          }, 
          {
            "familyName": "Sharma", 
            "givenName": "K R", 
            "id": "sg:person.01201204043.85", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01201204043.85"
            ], 
            "type": "Person"
          }, 
          {
            "familyName": "Miller", 
            "givenName": "R G", 
            "id": "sg:person.011536701204.29", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.011536701204.29"
            ], 
            "type": "Person"
          }, 
          {
            "familyName": "Steinman", 
            "givenName": "L", 
            "id": "sg:person.01317762565.74", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01317762565.74"
            ], 
            "type": "Person"
          }, 
          {
            "familyName": "Blau", 
            "givenName": "H M", 
            "id": "sg:person.01307671071.44", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01307671071.44"
            ], 
            "type": "Person"
          }
        ], 
        "citation": [
          {
            "id": "https://doi.org/10.1056/nejm198901193200302", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1001787997"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0014-5793(90)80199-s", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1002207519"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/bf00205170", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1007372734", 
              "https://doi.org/10.1007/bf00205170"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/bf00205170", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1007372734", 
              "https://doi.org/10.1007/bf00205170"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/mus.880121013", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1009990205"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0140-6736(90)93305-9", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1013203184"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0140-6736(90)93305-9", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1013203184"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1093/nar/16.23.11141", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1015096617"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0092-8674(92)90198-l", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1016505733"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0092-8674(87)90504-6", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1020401608"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0022-510x(90)90195-s", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1020963253"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0022-510x(90)90195-s", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1020963253"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/333858a0", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1024149606", 
              "https://doi.org/10.1038/333858a0"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1056/nejm198805263182104", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1025119452"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/352815a0", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1028509594", 
              "https://doi.org/10.1038/352815a0"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0140-6736(90)91628-n", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1034973369"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0140-6736(90)91628-n", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1034973369"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/337176a0", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1036011978", 
              "https://doi.org/10.1038/337176a0"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/329638a0", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1036706137", 
              "https://doi.org/10.1038/329638a0"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/345350a0", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1037000148", 
              "https://doi.org/10.1038/345350a0"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0896-6273(89)90226-2", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1040742379"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/0092-8674(87)90579-4", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1041056645"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/mus.880140302", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1048515004"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1006/abio.1987.9999", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1054484911"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1038/328434a0", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1056438814", 
              "https://doi.org/10.1038/328434a0"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1126/science.1962212", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1062515387"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1126/science.1962213", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1062515388"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://app.dimensions.ai/details/publication/pub.1078791147", 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://app.dimensions.ai/details/publication/pub.1079203819", 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://app.dimensions.ai/details/publication/pub.1079531279", 
            "type": "CreativeWork"
          }
        ], 
        "datePublished": "1992-04", 
        "datePublishedReg": "1992-04-01", 
        "description": "Gene delivery by transplantation of normal myoblasts has been proposed as a treatment of the primary defect, lack of the muscle protein dystrophin, that causes Duchenne muscular dystrophy (DMD), a lethal human muscle degenerative disorder. To test this possibility, we transplanted normal myoblasts from a father or an unaffected sibling into the muscle of eight boys with DMD, and assessed their production of dystrophin. Three patients with deletions in the dystrophin gene expressed normal dystrophin transcripts in muscle biopsy specimens taken from the transplant site one month after myoblast injection. Using the polymerase chain reaction we established that the dystrophin in these biopsies derived from donor myoblast DNA. These results show that transplanted myoblasts persist and produce dystrophin in muscle fibres of DMD patients.", 
        "genre": "research_article", 
        "id": "sg:pub.10.1038/356435a0", 
        "inLanguage": [
          "en"
        ], 
        "isAccessibleForFree": false, 
        "isPartOf": [
          {
            "id": "sg:journal.1018957", 
            "issn": [
              "0090-0028", 
              "1476-4687"
            ], 
            "name": "Nature", 
            "type": "Periodical"
          }, 
          {
            "issueNumber": "6368", 
            "type": "PublicationIssue"
          }, 
          {
            "type": "PublicationVolume", 
            "volumeNumber": "356"
          }
        ], 
        "name": "Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation", 
        "pagination": "435-438", 
        "productId": [
          {
            "name": "readcube_id", 
            "type": "PropertyValue", 
            "value": [
              "7aebe0347534fc29f70e36715c1fe51662a604bb5d2d85ab4ed477c24ba04909"
            ]
          }, 
          {
            "name": "pubmed_id", 
            "type": "PropertyValue", 
            "value": [
              "1557125"
            ]
          }, 
          {
            "name": "nlm_unique_id", 
            "type": "PropertyValue", 
            "value": [
              "0410462"
            ]
          }, 
          {
            "name": "doi", 
            "type": "PropertyValue", 
            "value": [
              "10.1038/356435a0"
            ]
          }, 
          {
            "name": "dimensions_id", 
            "type": "PropertyValue", 
            "value": [
              "pub.1025801133"
            ]
          }
        ], 
        "sameAs": [
          "https://doi.org/10.1038/356435a0", 
          "https://app.dimensions.ai/details/publication/pub.1025801133"
        ], 
        "sdDataset": "articles", 
        "sdDatePublished": "2019-04-10T15:38", 
        "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/v90/0000000001_0000000264/records_8664_00000424.jsonl", 
        "type": "ScholarlyArticle", 
        "url": "http://www.nature.com/articles/356435a0"
      }
    ]
     

    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.10.1038/356435a0'

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

    curl -H 'Accept: application/n-triples' 'https://scigraph.springernature.com/pub.10.1038/356435a0'

    Turtle is a human-readable linked data format.

    curl -H 'Accept: text/turtle' 'https://scigraph.springernature.com/pub.10.1038/356435a0'

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

    curl -H 'Accept: application/rdf+xml' 'https://scigraph.springernature.com/pub.10.1038/356435a0'


     

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

    251 TRIPLES      21 PREDICATES      71 URIs      37 LITERALS      25 BLANK NODES

    Subject Predicate Object
    1 sg:pub.10.1038/356435a0 schema:about N27bce98285174b7fbd620bfcefe21a95
    2 N2b4a787f31e04cc1a7178c4b849f54bc
    3 N6a54efc5c195400ba907b94b4b5ca0b3
    4 N6ac5f0d1851e45fe8e661b4e49fc2b75
    5 N72c88103e4d14a688ee75660fe564dcf
    6 N777a0056089d426b80821f7891e3ae2e
    7 N859917427a50445282ca7d62ff243b54
    8 N89af0f3ccfef4e06ba05034bd781d984
    9 N8a1afa9e2e6c42cfbfc44513e9b65ce8
    10 Nb03eeb1a9595413b86646fc01a01115b
    11 Nb1103638db6745969152dd61c57408d7
    12 Nb6fe6241508b43deaca49ef3f9772d68
    13 Nc0b6a82baf464e4f950c1b2cd4e476a6
    14 Nc3203b63ae2042108d6898ccb1e39829
    15 Nf2692d57e2f14867b969f12fcb6f9877
    16 Nfff26f15b0ef493d8898017171c09117
    17 anzsrc-for:10
    18 anzsrc-for:1004
    19 schema:author Nca0ecb1006bf42e99fe0900a24289abc
    20 schema:citation sg:pub.10.1007/bf00205170
    21 sg:pub.10.1038/328434a0
    22 sg:pub.10.1038/329638a0
    23 sg:pub.10.1038/333858a0
    24 sg:pub.10.1038/337176a0
    25 sg:pub.10.1038/345350a0
    26 sg:pub.10.1038/352815a0
    27 https://app.dimensions.ai/details/publication/pub.1078791147
    28 https://app.dimensions.ai/details/publication/pub.1079203819
    29 https://app.dimensions.ai/details/publication/pub.1079531279
    30 https://doi.org/10.1002/mus.880121013
    31 https://doi.org/10.1002/mus.880140302
    32 https://doi.org/10.1006/abio.1987.9999
    33 https://doi.org/10.1016/0014-5793(90)80199-s
    34 https://doi.org/10.1016/0022-510x(90)90195-s
    35 https://doi.org/10.1016/0092-8674(87)90504-6
    36 https://doi.org/10.1016/0092-8674(87)90579-4
    37 https://doi.org/10.1016/0092-8674(92)90198-l
    38 https://doi.org/10.1016/0140-6736(90)91628-n
    39 https://doi.org/10.1016/0140-6736(90)93305-9
    40 https://doi.org/10.1016/0896-6273(89)90226-2
    41 https://doi.org/10.1056/nejm198805263182104
    42 https://doi.org/10.1056/nejm198901193200302
    43 https://doi.org/10.1093/nar/16.23.11141
    44 https://doi.org/10.1126/science.1962212
    45 https://doi.org/10.1126/science.1962213
    46 schema:datePublished 1992-04
    47 schema:datePublishedReg 1992-04-01
    48 schema:description Gene delivery by transplantation of normal myoblasts has been proposed as a treatment of the primary defect, lack of the muscle protein dystrophin, that causes Duchenne muscular dystrophy (DMD), a lethal human muscle degenerative disorder. To test this possibility, we transplanted normal myoblasts from a father or an unaffected sibling into the muscle of eight boys with DMD, and assessed their production of dystrophin. Three patients with deletions in the dystrophin gene expressed normal dystrophin transcripts in muscle biopsy specimens taken from the transplant site one month after myoblast injection. Using the polymerase chain reaction we established that the dystrophin in these biopsies derived from donor myoblast DNA. These results show that transplanted myoblasts persist and produce dystrophin in muscle fibres of DMD patients.
    49 schema:genre research_article
    50 schema:inLanguage en
    51 schema:isAccessibleForFree false
    52 schema:isPartOf N83548b4115764846bbfce52a5638429d
    53 Nb8563002e40b4abebcea52939912e230
    54 sg:journal.1018957
    55 schema:name Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation
    56 schema:pagination 435-438
    57 schema:productId N2550c12eb0cf4d14875cd3c1bae4408c
    58 N352965f46d2d47da82aab9c281673a7b
    59 N4489923dbd0f44f89c8690bfd7102600
    60 N48a9dc18e4274db6995547cddfb13ab0
    61 Nccf70fecf29f4c409f6d4071075d9839
    62 schema:sameAs https://app.dimensions.ai/details/publication/pub.1025801133
    63 https://doi.org/10.1038/356435a0
    64 schema:sdDatePublished 2019-04-10T15:38
    65 schema:sdLicense https://scigraph.springernature.com/explorer/license/
    66 schema:sdPublisher N2db41e4873854477a2968405f7a4db06
    67 schema:url http://www.nature.com/articles/356435a0
    68 sgo:license sg:explorer/license/
    69 sgo:sdDataset articles
    70 rdf:type schema:ScholarlyArticle
    71 N22e37c7b21b34b65a9f73ec81cb0f223 rdf:first sg:person.01201204043.85
    72 rdf:rest Nef60ab2047b54fa78e74c54bf44dc931
    73 N2550c12eb0cf4d14875cd3c1bae4408c schema:name doi
    74 schema:value 10.1038/356435a0
    75 rdf:type schema:PropertyValue
    76 N27bce98285174b7fbd620bfcefe21a95 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    77 schema:name Adult
    78 rdf:type schema:DefinedTerm
    79 N2b4a787f31e04cc1a7178c4b849f54bc schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    80 schema:name Transcription, Genetic
    81 rdf:type schema:DefinedTerm
    82 N2db41e4873854477a2968405f7a4db06 schema:name Springer Nature - SN SciGraph project
    83 rdf:type schema:Organization
    84 N352965f46d2d47da82aab9c281673a7b schema:name pubmed_id
    85 schema:value 1557125
    86 rdf:type schema:PropertyValue
    87 N4489923dbd0f44f89c8690bfd7102600 schema:name dimensions_id
    88 schema:value pub.1025801133
    89 rdf:type schema:PropertyValue
    90 N48a9dc18e4274db6995547cddfb13ab0 schema:name readcube_id
    91 schema:value 7aebe0347534fc29f70e36715c1fe51662a604bb5d2d85ab4ed477c24ba04909
    92 rdf:type schema:PropertyValue
    93 N583347a0726b4cb2976efad6bb15abc1 rdf:first sg:person.01223601511.47
    94 rdf:rest Nc5628f430b7f4bfeb83550d24769c813
    95 N6a54efc5c195400ba907b94b4b5ca0b3 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    96 schema:name Oligonucleotide Probes
    97 rdf:type schema:DefinedTerm
    98 N6ac5f0d1851e45fe8e661b4e49fc2b75 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    99 schema:name Polymerase Chain Reaction
    100 rdf:type schema:DefinedTerm
    101 N72c88103e4d14a688ee75660fe564dcf schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    102 schema:name Oligodeoxyribonucleotides
    103 rdf:type schema:DefinedTerm
    104 N777a0056089d426b80821f7891e3ae2e schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    105 schema:name RNA, Messenger
    106 rdf:type schema:DefinedTerm
    107 N83548b4115764846bbfce52a5638429d schema:issueNumber 6368
    108 rdf:type schema:PublicationIssue
    109 N859917427a50445282ca7d62ff243b54 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    110 schema:name Molecular Sequence Data
    111 rdf:type schema:DefinedTerm
    112 N89af0f3ccfef4e06ba05034bd781d984 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    113 schema:name Dystrophin
    114 rdf:type schema:DefinedTerm
    115 N8a1afa9e2e6c42cfbfc44513e9b65ce8 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    116 schema:name Base Sequence
    117 rdf:type schema:DefinedTerm
    118 Nb03eeb1a9595413b86646fc01a01115b schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    119 schema:name Humans
    120 rdf:type schema:DefinedTerm
    121 Nb1103638db6745969152dd61c57408d7 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    122 schema:name Child
    123 rdf:type schema:DefinedTerm
    124 Nb6fe6241508b43deaca49ef3f9772d68 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    125 schema:name HLA Antigens
    126 rdf:type schema:DefinedTerm
    127 Nb8563002e40b4abebcea52939912e230 schema:volumeNumber 356
    128 rdf:type schema:PublicationVolume
    129 Nc0b6a82baf464e4f950c1b2cd4e476a6 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    130 schema:name Muscles
    131 rdf:type schema:DefinedTerm
    132 Nc3203b63ae2042108d6898ccb1e39829 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    133 schema:name Exons
    134 rdf:type schema:DefinedTerm
    135 Nc5628f430b7f4bfeb83550d24769c813 rdf:first sg:person.01150213550.00
    136 rdf:rest N22e37c7b21b34b65a9f73ec81cb0f223
    137 Nca0ecb1006bf42e99fe0900a24289abc rdf:first sg:person.0605476717.51
    138 rdf:rest N583347a0726b4cb2976efad6bb15abc1
    139 Nccf70fecf29f4c409f6d4071075d9839 schema:name nlm_unique_id
    140 schema:value 0410462
    141 rdf:type schema:PropertyValue
    142 Nef60ab2047b54fa78e74c54bf44dc931 rdf:first sg:person.011536701204.29
    143 rdf:rest Nfb9b6f81b83d49f5aa93430fe1602a65
    144 Nf2692d57e2f14867b969f12fcb6f9877 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    145 schema:name Muscular Dystrophies
    146 rdf:type schema:DefinedTerm
    147 Nfb9b6f81b83d49f5aa93430fe1602a65 rdf:first sg:person.01317762565.74
    148 rdf:rest Nfdd8b8b19dfa4be48eefa7c44b7d4e8a
    149 Nfdd8b8b19dfa4be48eefa7c44b7d4e8a rdf:first sg:person.01307671071.44
    150 rdf:rest rdf:nil
    151 Nfff26f15b0ef493d8898017171c09117 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
    152 schema:name Histocompatibility Testing
    153 rdf:type schema:DefinedTerm
    154 anzsrc-for:10 schema:inDefinedTermSet anzsrc-for:
    155 schema:name Technology
    156 rdf:type schema:DefinedTerm
    157 anzsrc-for:1004 schema:inDefinedTermSet anzsrc-for:
    158 schema:name Medical Biotechnology
    159 rdf:type schema:DefinedTerm
    160 sg:journal.1018957 schema:issn 0090-0028
    161 1476-4687
    162 schema:name Nature
    163 rdf:type schema:Periodical
    164 sg:person.01150213550.00 schema:familyName Lanctot
    165 schema:givenName A M
    166 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01150213550.00
    167 rdf:type schema:Person
    168 sg:person.011536701204.29 schema:familyName Miller
    169 schema:givenName R G
    170 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.011536701204.29
    171 rdf:type schema:Person
    172 sg:person.01201204043.85 schema:familyName Sharma
    173 schema:givenName K R
    174 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01201204043.85
    175 rdf:type schema:Person
    176 sg:person.01223601511.47 schema:familyName Pavlath
    177 schema:givenName G K
    178 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01223601511.47
    179 rdf:type schema:Person
    180 sg:person.01307671071.44 schema:familyName Blau
    181 schema:givenName H M
    182 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01307671071.44
    183 rdf:type schema:Person
    184 sg:person.01317762565.74 schema:familyName Steinman
    185 schema:givenName L
    186 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01317762565.74
    187 rdf:type schema:Person
    188 sg:person.0605476717.51 schema:affiliation https://www.grid.ac/institutes/grid.168010.e
    189 schema:familyName Gussoni
    190 schema:givenName E
    191 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0605476717.51
    192 rdf:type schema:Person
    193 sg:pub.10.1007/bf00205170 schema:sameAs https://app.dimensions.ai/details/publication/pub.1007372734
    194 https://doi.org/10.1007/bf00205170
    195 rdf:type schema:CreativeWork
    196 sg:pub.10.1038/328434a0 schema:sameAs https://app.dimensions.ai/details/publication/pub.1056438814
    197 https://doi.org/10.1038/328434a0
    198 rdf:type schema:CreativeWork
    199 sg:pub.10.1038/329638a0 schema:sameAs https://app.dimensions.ai/details/publication/pub.1036706137
    200 https://doi.org/10.1038/329638a0
    201 rdf:type schema:CreativeWork
    202 sg:pub.10.1038/333858a0 schema:sameAs https://app.dimensions.ai/details/publication/pub.1024149606
    203 https://doi.org/10.1038/333858a0
    204 rdf:type schema:CreativeWork
    205 sg:pub.10.1038/337176a0 schema:sameAs https://app.dimensions.ai/details/publication/pub.1036011978
    206 https://doi.org/10.1038/337176a0
    207 rdf:type schema:CreativeWork
    208 sg:pub.10.1038/345350a0 schema:sameAs https://app.dimensions.ai/details/publication/pub.1037000148
    209 https://doi.org/10.1038/345350a0
    210 rdf:type schema:CreativeWork
    211 sg:pub.10.1038/352815a0 schema:sameAs https://app.dimensions.ai/details/publication/pub.1028509594
    212 https://doi.org/10.1038/352815a0
    213 rdf:type schema:CreativeWork
    214 https://app.dimensions.ai/details/publication/pub.1078791147 schema:CreativeWork
    215 https://app.dimensions.ai/details/publication/pub.1079203819 schema:CreativeWork
    216 https://app.dimensions.ai/details/publication/pub.1079531279 schema:CreativeWork
    217 https://doi.org/10.1002/mus.880121013 schema:sameAs https://app.dimensions.ai/details/publication/pub.1009990205
    218 rdf:type schema:CreativeWork
    219 https://doi.org/10.1002/mus.880140302 schema:sameAs https://app.dimensions.ai/details/publication/pub.1048515004
    220 rdf:type schema:CreativeWork
    221 https://doi.org/10.1006/abio.1987.9999 schema:sameAs https://app.dimensions.ai/details/publication/pub.1054484911
    222 rdf:type schema:CreativeWork
    223 https://doi.org/10.1016/0014-5793(90)80199-s schema:sameAs https://app.dimensions.ai/details/publication/pub.1002207519
    224 rdf:type schema:CreativeWork
    225 https://doi.org/10.1016/0022-510x(90)90195-s schema:sameAs https://app.dimensions.ai/details/publication/pub.1020963253
    226 rdf:type schema:CreativeWork
    227 https://doi.org/10.1016/0092-8674(87)90504-6 schema:sameAs https://app.dimensions.ai/details/publication/pub.1020401608
    228 rdf:type schema:CreativeWork
    229 https://doi.org/10.1016/0092-8674(87)90579-4 schema:sameAs https://app.dimensions.ai/details/publication/pub.1041056645
    230 rdf:type schema:CreativeWork
    231 https://doi.org/10.1016/0092-8674(92)90198-l schema:sameAs https://app.dimensions.ai/details/publication/pub.1016505733
    232 rdf:type schema:CreativeWork
    233 https://doi.org/10.1016/0140-6736(90)91628-n schema:sameAs https://app.dimensions.ai/details/publication/pub.1034973369
    234 rdf:type schema:CreativeWork
    235 https://doi.org/10.1016/0140-6736(90)93305-9 schema:sameAs https://app.dimensions.ai/details/publication/pub.1013203184
    236 rdf:type schema:CreativeWork
    237 https://doi.org/10.1016/0896-6273(89)90226-2 schema:sameAs https://app.dimensions.ai/details/publication/pub.1040742379
    238 rdf:type schema:CreativeWork
    239 https://doi.org/10.1056/nejm198805263182104 schema:sameAs https://app.dimensions.ai/details/publication/pub.1025119452
    240 rdf:type schema:CreativeWork
    241 https://doi.org/10.1056/nejm198901193200302 schema:sameAs https://app.dimensions.ai/details/publication/pub.1001787997
    242 rdf:type schema:CreativeWork
    243 https://doi.org/10.1093/nar/16.23.11141 schema:sameAs https://app.dimensions.ai/details/publication/pub.1015096617
    244 rdf:type schema:CreativeWork
    245 https://doi.org/10.1126/science.1962212 schema:sameAs https://app.dimensions.ai/details/publication/pub.1062515387
    246 rdf:type schema:CreativeWork
    247 https://doi.org/10.1126/science.1962213 schema:sameAs https://app.dimensions.ai/details/publication/pub.1062515388
    248 rdf:type schema:CreativeWork
    249 https://www.grid.ac/institutes/grid.168010.e schema:alternateName Stanford University
    250 schema:name Department of Neurology and Neurological Sciences, Stanford University School of Medicine, California 94305.
    251 rdf:type schema:Organization
     




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


    ...