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Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model.
Seo, Young-Eun; Baine, Stephen H; Kempton, Amber N; Rogers, Oliver C; Lewis, Sarah; Adegboye, Kaitlin; Haile, Alex; Griffin, Danielle A; Peterson, Ellyn L; Pozsgai, Eric R; Potter, Rachael A; Rodino-Klapac, Louise R.
Affiliation
  • Seo YE; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Baine SH; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Kempton AN; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Rogers OC; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Lewis S; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Adegboye K; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Haile A; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Griffin DA; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Peterson EL; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Pozsgai ER; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Potter RA; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Rodino-Klapac LR; Research & Development, Gene Therapy Research, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Mol Ther Methods Clin Dev ; 28: 284-299, 2023 Mar 09.
Article in En | MEDLINE | ID: mdl-36816759
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Full text: 1 Database: MEDLINE Language: En Year: 2023 Type: Article
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Full text: 1 Database: MEDLINE Language: En Year: 2023 Type: Article