{"id":"https://openalex.org/W2889510460","doi":"https://doi.org/10.1126/science.aau1549","title":"Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy","display_name":"Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy","publication_year":2018,"publication_date":"2018-08-30","ids":{"openalex":"https://openalex.org/W2889510460","doi":"https://doi.org/10.1126/science.aau1549","mag":"2889510460","pmid":"https://pubmed.ncbi.nlm.nih.gov/30166439","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/6205228"},"language":"en","primary_location":{"id":"doi:10.1126/science.aau1549","doi":"https://doi.org/10.1126/science.aau1549","is_oa":false,"landing_page_url":"https://doi.org/10.1126/science.aau1549","pdf_url":null,"source":{"id":"https://openalex.org/S3880285","display_name":"Science","issn_l":"0036-8075","issn":["0036-8075","0193-4511","1095-9203"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","erih-plus","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310315823","host_organization_name":"American Association for the Advancement of Science","host_organization_lineage":["https://openalex.org/P4310315823"],"host_organization_lineage_names":["American Association for the Advancement of Science"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Science","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"http://europepmc.org/pmc/articles/PMC6205228","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5028726673","display_name":"Leonela Amoasii","orcid":"https://orcid.org/0000-0001-7960-2749","observed_orcids":["https://orcid.org/0000-0001-7960-2749"]},"institutions":[{"id":"https://openalex.org/I867280407","display_name":"The University of Texas Southwestern Medical Center","ror":"https://ror.org/05byvp690","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I867280407"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Leonela Amoasii","raw_affiliation_strings":["Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","Exonics Therapeutics, 75 Kneeland Street, Boston, MA 02111, USA"],"raw_orcid":"https://orcid.org/0000-0001-7960-2749","affiliations":[{"raw_affiliation_string":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","institution_ids":["https://openalex.org/I867280407"]},{"raw_affiliation_string":"Exonics Therapeutics, 75 Kneeland Street, Boston, MA 02111, USA","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5042740304","display_name":"John C. W. Hildyard","orcid":"https://orcid.org/0000-0003-2283-2118","observed_orcids":["https://orcid.org/0000-0003-2283-2118"]},"institutions":[{"id":"https://openalex.org/I133836048","display_name":"Royal Veterinary College","ror":"https://ror.org/01wka8n18","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I133836048"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"John C. W. Hildyard","raw_affiliation_strings":["Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK"],"raw_orcid":"https://orcid.org/0000-0003-2283-2118","affiliations":[{"raw_affiliation_string":"Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK","institution_ids":["https://openalex.org/I133836048"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5100423886","display_name":"Hui zi Li","orcid":"https://orcid.org/0000-0002-4114-8349","observed_orcids":["https://orcid.org/0000-0002-4114-8349"]},"institutions":[{"id":"https://openalex.org/I867280407","display_name":"The University of Texas Southwestern Medical Center","ror":"https://ror.org/05byvp690","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I867280407"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Hui Li","raw_affiliation_strings":["Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA"],"raw_orcid":"https://orcid.org/0000-0002-4114-8349","affiliations":[{"raw_affiliation_string":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","institution_ids":["https://openalex.org/I867280407"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5085694146","display_name":"Efrain Sanchez\u2010Ortiz","orcid":"https://orcid.org/0000-0001-9358-1630","observed_orcids":["https://orcid.org/0000-0001-9358-1630"]},"institutions":[{"id":"https://openalex.org/I867280407","display_name":"The University of Texas Southwestern Medical Center","ror":"https://ror.org/05byvp690","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I867280407"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Efrain Sanchez-Ortiz","raw_affiliation_strings":["Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA"],"raw_orcid":"https://orcid.org/0000-0001-9358-1630","affiliations":[{"raw_affiliation_string":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","institution_ids":["https://openalex.org/I867280407"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5020015079","display_name":"Alex A. Mireault","orcid":"https://orcid.org/0000-0002-7534-636X","observed_orcids":["https://orcid.org/0000-0002-7534-636X"]},"institutions":[{"id":"https://openalex.org/I867280407","display_name":"The University of Texas Southwestern Medical Center","ror":"https://ror.org/05byvp690","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I867280407"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Alex Mireault","raw_affiliation_strings":["Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA"],"raw_orcid":"https://orcid.org/0000-0002-7534-636X","affiliations":[{"raw_affiliation_string":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","institution_ids":["https://openalex.org/I867280407"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5064702820","display_name":"Daniel Caballero","orcid":"https://orcid.org/0000-0001-9232-8991","observed_orcids":["https://orcid.org/0000-0001-9232-8991"]},"institutions":[{"id":"https://openalex.org/I867280407","display_name":"The University of Texas Southwestern Medical Center","ror":"https://ror.org/05byvp690","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I867280407"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Daniel Caballero","raw_affiliation_strings":["Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA"],"raw_orcid":"https://orcid.org/0000-0001-9232-8991","affiliations":[{"raw_affiliation_string":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","institution_ids":["https://openalex.org/I867280407"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5070098311","display_name":"Rachel C. M. Harron","orcid":"https://orcid.org/0000-0001-9418-0708","observed_orcids":["https://orcid.org/0000-0001-9418-0708"]},"institutions":[{"id":"https://openalex.org/I133836048","display_name":"Royal Veterinary College","ror":"https://ror.org/01wka8n18","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I133836048"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Rachel Harron","raw_affiliation_strings":["Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK"],"raw_orcid":"https://orcid.org/0000-0001-9418-0708","affiliations":[{"raw_affiliation_string":"Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK","institution_ids":["https://openalex.org/I133836048"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5079615980","display_name":"Thaleia\u2010Rengina Stathopoulou","orcid":"https://orcid.org/0000-0003-4666-2002","observed_orcids":["https://orcid.org/0000-0003-4666-2002"]},"institutions":[{"id":"https://openalex.org/I133836048","display_name":"Royal Veterinary College","ror":"https://ror.org/01wka8n18","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I133836048"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Thaleia-Rengina Stathopoulou","raw_affiliation_strings":["Section of Anaesthesia and Analgesia, Royal Veterinary College, London NW1 0TU, UK"],"raw_orcid":"https://orcid.org/0000-0003-4666-2002","affiliations":[{"raw_affiliation_string":"Section of Anaesthesia and Analgesia, Royal Veterinary College, London NW1 0TU, UK","institution_ids":["https://openalex.org/I133836048"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5031969874","display_name":"Claire Massey","orcid":"https://orcid.org/0000-0003-0823-6547","observed_orcids":["https://orcid.org/0000-0003-0823-6547"]},"institutions":[{"id":"https://openalex.org/I133836048","display_name":"Royal Veterinary College","ror":"https://ror.org/01wka8n18","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I133836048"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Claire Massey","raw_affiliation_strings":["Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK"],"raw_orcid":"https://orcid.org/0000-0003-0823-6547","affiliations":[{"raw_affiliation_string":"Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK","institution_ids":["https://openalex.org/I133836048"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5074600638","display_name":"John Michael Shelton","orcid":"https://orcid.org/0000-0002-2949-1638","observed_orcids":["https://orcid.org/0000-0002-2949-1638"]},"institutions":[{"id":"https://openalex.org/I867280407","display_name":"The University of Texas Southwestern Medical Center","ror":"https://ror.org/05byvp690","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I867280407"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"John M. Shelton","raw_affiliation_strings":["Department of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA"],"raw_orcid":"https://orcid.org/0000-0002-2949-1638","affiliations":[{"raw_affiliation_string":"Department of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","institution_ids":["https://openalex.org/I867280407"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5024308650","display_name":"Rhonda Bassel\u2010Duby","orcid":"https://orcid.org/0000-0001-7568-410X","observed_orcids":["https://orcid.org/0000-0001-7568-410X"]},"institutions":[{"id":"https://openalex.org/I867280407","display_name":"The University of Texas Southwestern Medical Center","ror":"https://ror.org/05byvp690","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I867280407"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Rhonda Bassel-Duby","raw_affiliation_strings":["Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA"],"raw_orcid":"https://orcid.org/0000-0001-7568-410X","affiliations":[{"raw_affiliation_string":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","institution_ids":["https://openalex.org/I867280407"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5089252543","display_name":"Richard J. Piercy","orcid":"https://orcid.org/0000-0002-4344-6438","observed_orcids":["https://orcid.org/0000-0002-4344-6438"]},"institutions":[{"id":"https://openalex.org/I133836048","display_name":"Royal Veterinary College","ror":"https://ror.org/01wka8n18","country_code":"GB","type":"education","lineage":["https://openalex.org/I124357947","https://openalex.org/I133836048"]}],"countries":["GB"],"is_corresponding":false,"raw_author_name":"Richard J. Piercy","raw_affiliation_strings":["Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK"],"raw_orcid":"https://orcid.org/0000-0002-4344-6438","affiliations":[{"raw_affiliation_string":"Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK","institution_ids":["https://openalex.org/I133836048"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5084182062","display_name":"Eric N. Olson","orcid":"https://orcid.org/0000-0003-1151-8262","observed_orcids":["https://orcid.org/0000-0003-1151-8262","https://orcid.org/0000-0002-8716-9532"]},"institutions":[{"id":"https://openalex.org/I867280407","display_name":"The University of Texas Southwestern Medical Center","ror":"https://ror.org/05byvp690","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I867280407"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Eric N. Olson","raw_affiliation_strings":["Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA"],"raw_orcid":"https://orcid.org/0000-0003-1151-8262","affiliations":[{"raw_affiliation_string":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA","institution_ids":["https://openalex.org/I867280407"]}]}],"institutions":[],"countries_distinct_count":2,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5084182062"],"corresponding_institution_ids":["https://openalex.org/I867280407"],"apc_list":null,"apc_paid":null,"fwci":23.336,"has_fulltext":false,"cited_by_count":564,"citation_normalized_percentile":{"value":0.99801095,"is_in_top_1_percent":true,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":88,"max":100},"biblio":{"volume":"362","issue":"6410","first_page":"86","last_page":"91"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10441","display_name":"Muscle Physiology and Disorders","score":0.6083999872207642,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10441","display_name":"Muscle Physiology and Disorders","score":0.6083999872207642,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10878","display_name":"CRISPR and Genetic Engineering","score":0.3075999915599823,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.02930000051856041,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/duchenne-muscular-dystrophy","display_name":"Duchenne muscular dystrophy","score":1.0},{"id":"https://openalex.org/keywords/dystrophin","display_name":"dystrophin","score":0.7829999923706055},{"id":"https://openalex.org/keywords/crispr-cas9-genome-editing","display_name":"CRISPR/Cas9 genome editing","score":0.9729999899864197},{"id":"https://openalex.org/keywords/canine-model","display_name":"canine model","score":0.871999979019165},{"id":"https://openalex.org/keywords/intramuscular-administration","display_name":"intramuscular administration","score":0.8489999771118164},{"id":"https://openalex.org/keywords/systemic-drug-delivery","display_name":"systemic drug delivery","score":0.9160000085830688},{"id":"https://openalex.org/keywords/skeletal-muscle","display_name":"skeletal muscle","score":0.8690000176429749},{"id":"https://openalex.org/keywords/myocardium","display_name":"myocardium","score":0.8230000138282776},{"id":"https://openalex.org/keywords/muscle-histology","display_name":"muscle histology","score":0.9629999995231628}],"concepts":[{"id":"https://openalex.org/C2778943923","wikidata":"https://www.wikidata.org/wiki/Q1648484","display_name":"Duchenne muscular dystrophy","level":2,"score":0.9216528534889221},{"id":"https://openalex.org/C2780394045","wikidata":"https://www.wikidata.org/wiki/Q412285","display_name":"Dystrophin","level":3,"score":0.7156646847724915},{"id":"https://openalex.org/C2779030066","wikidata":"https://www.wikidata.org/wiki/Q1137767","display_name":"Muscular dystrophy","level":2,"score":0.5209442377090454},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.49880409240722656},{"id":"https://openalex.org/C144501496","wikidata":"https://www.wikidata.org/wiki/Q5533489","display_name":"Genome editing","level":4,"score":0.4767671227455139},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.46361714601516724},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.4047284722328186},{"id":"https://openalex.org/C98108389","wikidata":"https://www.wikidata.org/wiki/Q412563","display_name":"CRISPR","level":3,"score":0.19332239031791687}],"mesh":[{"descriptor_ui":"D000072669","descriptor_name":"Gene Editing","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":true},{"descriptor_ui":"D000256","descriptor_name":"Adenoviridae","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004195","descriptor_name":"Disease Models, Animal","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004285","descriptor_name":"Dogs","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D016189","descriptor_name":"Dystrophin","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D016189","descriptor_name":"Dystrophin","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D018014","descriptor_name":"Gene Transfer Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D020388","descriptor_name":"Muscular Dystrophy, Duchenne","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":true},{"descriptor_ui":"D064113","descriptor_name":"CRISPR-Cas Systems","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":5,"locations":[{"id":"doi:10.1126/science.aau1549","doi":"https://doi.org/10.1126/science.aau1549","is_oa":false,"landing_page_url":"https://doi.org/10.1126/science.aau1549","pdf_url":null,"source":{"id":"https://openalex.org/S3880285","display_name":"Science","issn_l":"0036-8075","issn":["0036-8075","0193-4511","1095-9203"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","erih-plus","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310315823","host_organization_name":"American Association for the Advancement of Science","host_organization_lineage":["https://openalex.org/P4310315823"],"host_organization_lineage_names":["American Association for the Advancement of Science"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Science","raw_type":"journal-article"},{"id":"pmid:30166439","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/30166439","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Science (New York, N.Y.)","raw_type":"Journal Article"},{"id":"pmh:oai:europepmc.org:5151232","doi":null,"is_oa":true,"landing_page_url":"http://europepmc.org/pmc/articles/PMC6205228","pdf_url":null,"source":{"id":"https://openalex.org/S4306400806","display_name":"Europe PMC (PubMed Central)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1303153112","host_organization_name":"European Bioinformatics Institute","host_organization_lineage":["https://openalex.org/I1303153112"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"","raw_type":"Text"},{"id":"pmh:oai:pubmedcentral.nih.gov:6205228","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/6205228","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Science","raw_type":"Text"},{"id":"pmh:oai:researchonline.rvc.ac.uk:11792","doi":null,"is_oa":true,"landing_page_url":"https://researchonline.rvc.ac.uk/id/eprint/11792/1/11792.pdf","pdf_url":"https://researchonline.rvc.ac.uk/id/eprint/11792/1/11792.pdf","source":{"id":"https://openalex.org/S4306402069","display_name":"RVC Research Online (Royal Veterinary College)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I133836048","host_organization_name":"Royal Veterinary College","host_organization_lineage":["https://openalex.org/I133836048"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"","raw_type":"Article"}],"best_oa_location":{"id":"pmh:oai:europepmc.org:5151232","doi":null,"is_oa":true,"landing_page_url":"http://europepmc.org/pmc/articles/PMC6205228","pdf_url":null,"source":{"id":"https://openalex.org/S4306400806","display_name":"Europe PMC (PubMed Central)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1303153112","host_organization_name":"European Bioinformatics Institute","host_organization_lineage":["https://openalex.org/I1303153112"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"","raw_type":"Text"},"sustainable_development_goals":[{"score":0.9074000120162964,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G1516416088","display_name":"Heart Repair through Reprogramming of Cardiac Cell Fate","funder_award_id":"R01HL077439","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G366698205","display_name":"Developing and phenotyping a colony of dogs with dystrophin-deficient muscular dystrophy: a strategic resource for UK-based research into Duchenne Muscular Dystrophy.","funder_award_id":"101550/Z/13/Z","funder_id":"https://openalex.org/F4320311904","funder_display_name":"Wellcome Trust"},{"id":"https://openalex.org/G662211534","display_name":null,"funder_award_id":"1\u20130025","funder_id":"https://openalex.org/F4320306196","funder_display_name":"Welch Foundation"},{"id":"https://openalex.org/G67469951","display_name":"Molecular Dissection of Myoblast Fusion In Muscle Development and Regeneration","funder_award_id":"R01AR067294","funder_id":"https://openalex.org/F4320337362","funder_display_name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases"},{"id":"https://openalex.org/G7114834284","display_name":"Deciphering the role of a novel micropeptide in cardiac function and dysfunction","funder_award_id":"R01HL130253","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G7166158189","display_name":"Metabolic Control by Myogenic MicroRNAs, Mediator and Myokines","funder_award_id":"R01DK099653","funder_id":"https://openalex.org/F4320337357","funder_display_name":"National Institute of Diabetes and Digestive and Kidney Diseases"},{"id":"https://openalex.org/G988446309","display_name":"Myoediting of Duchenne muscular dystrophy","funder_award_id":"U54HD087351","funder_id":"https://openalex.org/F4320337611","funder_display_name":"Eunice Kennedy Shriver National Institute of Child Health and Human Development"}],"funders":[{"id":"https://openalex.org/F4320306196","display_name":"Welch Foundation","ror":"https://ror.org/00np6vq88"},{"id":"https://openalex.org/F4320311904","display_name":"Wellcome Trust","ror":"https://ror.org/029chgv08"},{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320337338","display_name":"National Heart, Lung, and Blood Institute","ror":"https://ror.org/012pb6c26"},{"id":"https://openalex.org/F4320337357","display_name":"National Institute of Diabetes and Digestive and Kidney Diseases","ror":"https://ror.org/00adh9b73"},{"id":"https://openalex.org/F4320337362","display_name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases","ror":"https://ror.org/006zn3t30"},{"id":"https://openalex.org/F4320337611","display_name":"Eunice Kennedy Shriver National Institute of Child Health and Human Development","ror":"https://ror.org/04byxyr05"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":35,"referenced_works":["https://openalex.org/W147075648","https://openalex.org/W1936484224","https://openalex.org/W1987745724","https://openalex.org/W1997490017","https://openalex.org/W2008289270","https://openalex.org/W2008980136","https://openalex.org/W2018862827","https://openalex.org/W2033411843","https://openalex.org/W2044964290","https://openalex.org/W2059882448","https://openalex.org/W2072072113","https://openalex.org/W2081550861","https://openalex.org/W2100279229","https://openalex.org/W2101455790","https://openalex.org/W2106070783","https://openalex.org/W2111700646","https://openalex.org/W2129682060","https://openalex.org/W2147781659","https://openalex.org/W2196662084","https://openalex.org/W2203460689","https://openalex.org/W2208696160","https://openalex.org/W2234725241","https://openalex.org/W2257132471","https://openalex.org/W2283559138","https://openalex.org/W2494748207","https://openalex.org/W2554491985","https://openalex.org/W2560266955","https://openalex.org/W2560408974","https://openalex.org/W2587479628","https://openalex.org/W2588637030","https://openalex.org/W2765253110","https://openalex.org/W2775147596","https://openalex.org/W2793661486","https://openalex.org/W2801592524","https://openalex.org/W2883697525"],"related_works":["https://openalex.org/W2895067096","https://openalex.org/W2891352395","https://openalex.org/W2274923116","https://openalex.org/W2893054640","https://openalex.org/W2898855929","https://openalex.org/W2196662084","https://openalex.org/W2208696160","https://openalex.org/W3164247953","https://openalex.org/W4210920902","https://openalex.org/W2566046886"],"abstract_inverted_index":{"Gene":[0],"editing":[1,44,93],"and":[2,15,39,51,106],"muscular":[3,6],"dystrophy":[4,7],"Duchenne":[5],"(DMD)":[8],"is":[9,22],"characterized":[10],"by":[11,24,115],"progressive":[12],"muscle":[13,117],"weakness":[14],"a":[16,62,73,97],"shortened":[17],"life":[18],"span.":[19],"The":[20,42],"disease":[21],"caused":[23],"mutations":[25,50],"that":[26,78],"reduce":[27],"or":[28,87],"prevent":[29],"expression":[30,112],"of":[31,59,76,82,90,110],"dystrophin,":[32],"an":[33],"essential":[34],"structural":[35],"protein":[36,102],"in":[37,56,72,96,100,104],"skeletal":[38,105],"heart":[40,107],"muscle.":[41,108],"gene":[43,92],"technology":[45],"CRISPR-Cas9":[46],"can":[47],"correct":[48],"disease-causing":[49],"has":[52],"yielded":[53],"promising":[54],"results":[55],"mouse":[57],"models":[58],"DMD.":[60],"In":[61],"small,":[63],"short-term":[64],"study,":[65],"Amoasii":[66],"et":[67],"al.":[68],"tested":[69],"this":[70,121],"strategy":[71],"dog":[74],"model":[75],"DMD":[77],"exhibits":[79],"many":[80],"features":[81],"the":[83,91],"human":[84],"disease.":[85],"Intramuscular":[86],"systemic":[88],"delivery":[89],"components":[94],"resulted":[95],"substantial":[98],"increase":[99],"dystrophin":[101,111],"levels":[103],"Restoration":[109],"was":[113],"accompanied":[114],"improved":[116],"histology.":[118],"Science":[119],",":[120],"issue":[122],"p.":[123],"86":[124]},"counts_by_year":[{"year":2026,"cited_by_count":23},{"year":2025,"cited_by_count":44},{"year":2024,"cited_by_count":41},{"year":2023,"cited_by_count":64},{"year":2022,"cited_by_count":92},{"year":2021,"cited_by_count":93},{"year":2020,"cited_by_count":88},{"year":2019,"cited_by_count":102},{"year":2018,"cited_by_count":15},{"year":2016,"cited_by_count":1}],"updated_date":"2026-11-07T11:25:07.637180","created_date":"2018-09-07T00:00:00"}
