Ontology highlight
ABSTRACT:
SUBMITTER: Sahenk Z
PROVIDER: S-EPMC8413669 | biostudies-literature | 2021 Sep
REPOSITORIES: biostudies-literature
Sahenk Zarife Z Ozes Burcak B Murrey Darren D Myers Morgan M Moss Kyle K Yalvac Mehmet E ME Ridgley Alicia A Chen Lei L Mendell Jerry R JR
Molecular therapy. Methods & clinical development 20210624
Limb girdle muscular dystrophy (LGMD) 2A/R1, caused by mutations in the <i>CAPN3</i> gene and CAPN3 loss of function, is known to play a role in disease pathogenicity. In this study, AAVrh74.tMCK.CAPN3 was delivered systemically to two different age groups of CAPN3 knockout (KO) mice; each group included two treatment cohorts receiving low (1.17 × 10<sup>14</sup> vg/kg) and high (2.35 × 10<sup>14</sup> vg/kg) doses of the vector and untreated controls. Treatment efficacy was tested 20 weeks afte ...[more]