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A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Mendell, Jerry R; Sahenk, Zarife; Malik, Vinod; Gomez, Ana M; Flanigan, Kevin M; Lowes, Linda P; Alfano, Lindsay N; Berry, Katherine; Meadows, Eric; Lewis, Sarah; Braun, Lyndsey; Shontz, Kim; Rouhana, Maria; Clark, Kelly Reed; Rosales, Xiomara Q; Al-Zaidy, Samiah; Govoni, Alessandra; Rodino-Klapac, Louise R; Hogan, Mark J; Kaspar, Brian K.
Affiliation
  • Mendell JR; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [3] Department of Neurology, The Ohio State University, Columbus, Ohio, USA.
  • Sahenk Z; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [3] Department of Neurology, The Ohio State University, Columbus, Ohio, USA.
  • Malik V; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Gomez AM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Flanigan KM; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [3] Department of Neurology, The Ohio State University, Columbus, Ohio, USA.
  • Lowes LP; 1] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [2] Department of Physical Medicine and Rehabilitation, The Ohio State University, Columbus, Ohio, USA.
  • Alfano LN; 1] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [2] Department of Physical Medicine and Rehabilitation, The Ohio State University, Columbus, Ohio, USA.
  • Berry K; 1] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [2] Department of Physical Medicine and Rehabilitation, The Ohio State University, Columbus, Ohio, USA.
  • Meadows E; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Lewis S; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Braun L; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Shontz K; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Rouhana M; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Clark KR; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
  • Rosales XQ; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
  • Al-Zaidy S; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
  • Govoni A; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Rodino-Klapac LR; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
  • Hogan MJ; Department of Radiology, Vascular and Interventional Radiology, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Kaspar BK; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
Mol Ther ; 23(1): 192-201, 2015 Jan.
Article in En | MEDLINE | ID: mdl-25322757
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Full text: 1 Database: MEDLINE Main subject: Genetic Therapy / Dystrophin / Muscular Dystrophy, Duchenne / Follistatin-Related Proteins / Myostatin Limits: Adult / Humans / Male Language: En Year: 2015 Type: Article
Fulltext
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Full text: 1 Database: MEDLINE Main subject: Genetic Therapy / Dystrophin / Muscular Dystrophy, Duchenne / Follistatin-Related Proteins / Myostatin Limits: Adult / Humans / Male Language: En Year: 2015 Type: Article