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Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy.
Griffin, Danielle A; Pozsgai, Eric R; Heller, Kristin N; Potter, Rachael A; Peterson, Ellyn L; Rodino-Klapac, Louise R.
Affiliation
  • Griffin DA; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Pozsgai ER; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
  • Heller KN; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Potter RA; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
  • Peterson EL; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Rodino-Klapac LR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Hum Gene Ther ; 32(7-8): 390-404, 2021 04.
Article in En | MEDLINE | ID: mdl-33349138

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Muscular Dystrophies, Limb-Girdle / Sarcoglycanopathies Type of study: Prognostic_studies / Risk_factors_studies Limits: Animals / Humans Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2021 Document type: Article Affiliation country: United States Country of publication: United States

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Muscular Dystrophies, Limb-Girdle / Sarcoglycanopathies Type of study: Prognostic_studies / Risk_factors_studies Limits: Animals / Humans Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2021 Document type: Article Affiliation country: United States Country of publication: United States