Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1
Zarife Sahenk, Burçak Özeş, Darren Murrey, Morgan Myers, Kyle Moss, Mehmet E. Yalvaç, Alicia Ridgley, Lei Chen, Jerry R. Mendell
Abstract
gene replacement therapy improved the phenotype in the CAPN3 KO mouse model at both doses independent of age at the time of vector administration. The improvements were supported by an absence of cardiotoxicity, showing the efficacy and safety of the AAV.CAPN3 vector as a potential gene therapy for LGMDR1.
Topics & Concepts
ContractilityCardiotoxicityMuscular dystrophyLimb-girdle muscular dystrophySkeletal muscleInternal medicineMedicineDystrophyPhenotypeBiologyPathologyGeneToxicityGeneticsMuscle Physiology and DisordersCalpain Protease Function and RegulationViral Infections and Immunology Research