Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.

Potter, Rachael A

Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy. [electronic resource] - Human gene therapy 07 2018 - 749-762 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

1557-7422

10.1089/hum.2017.062 doi


Animals
DNA, Complementary--administration & dosage
Dependovirus--genetics
Disease Models, Animal
Dysferlin--administration & dosage
Gene Expression Regulation
Genetic Therapy
Genetic Vectors--therapeutic use
Humans
Male
Mice
Muscle, Skeletal
Muscular Dystrophies, Limb-Girdle--genetics
Mutation