AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models.

ABSTRACT:

Objective

Dysferlinopathies are a family of untreatable muscle disorders caused by mutations in the dysferlin gene. Lack of dysferlin protein results in progressive dystrophy with chronic muscle fiber loss, inflammation, fat replacement, and fibrosis; leading to deteriorating muscle weakness. The objective of this work is to demonstrate efficient and safe restoration of dysferlin expression following gene therapy treatment.

Methods

Traditional gene therapy is restricted by the packaging capacity limit of adeno-associated virus (AAV), however, use of a dual vector strategy allows for delivery of over-sized genes, including dysferlin. The two vector system (AAV.DYSF.DV) packages the dysferlin cDNA utilizing AAV serotype rh.74 through the use of two discrete vectors defined by

SUBMITTER: Sondergaard PC 

PROVIDER: S-EPMC4369275 | biostudies-literature | 2015 Mar

REPOSITORIES: biostudies-literature