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AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression.
Gicquel, Evelyne; Maizonnier, Natacha; Foltz, Steven J; Martin, William J; Bourg, Nathalie; Svinartchouk, Fedor; Charton, Karine; Beedle, Aaron M; Richard, Isabelle.
Affiliation
  • Gicquel E; INSERM, U951, INTEGRARE Research Unit, Généthon, Evry, F-91002, France.
  • Maizonnier N; INSERM, U951, INTEGRARE Research Unit, Généthon, Evry, F-91002, France.
  • Foltz SJ; Pharmaceutical & Biomedical Sciences, University of Georgia College of Pharmacy, Athens, GA 30602, USA.
  • Martin WJ; Animal Health Research Center, University of Georgia, Athens, GA 30602, USA.
  • Bourg N; INSERM, U951, INTEGRARE Research Unit, Généthon, Evry, F-91002, France.
  • Svinartchouk F; INSERM, U951, INTEGRARE Research Unit, Généthon, Evry, F-91002, France.
  • Charton K; INSERM, U951, INTEGRARE Research Unit, Généthon, Evry, F-91002, France.
  • Beedle AM; Pharmaceutical & Biomedical Sciences, University of Georgia College of Pharmacy, Athens, GA 30602, USA.
  • Richard I; Pharmaceutical Sciences, Binghamton University SUNY, Binghamton, NY 13902, USA.
Hum Mol Genet ; 26(10): 1952-1965, 2017 05 15.
Article in En | MEDLINE | ID: mdl-28334834
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Full text: 1 Database: MEDLINE Main subject: Proteins / Muscular Dystrophies, Limb-Girdle Limits: Animals Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2017 Type: Article Affiliation country: France
Fulltext
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Full text: 1 Database: MEDLINE Main subject: Proteins / Muscular Dystrophies, Limb-Girdle Limits: Animals Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2017 Type: Article Affiliation country: France