Article
Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.
Human gene therapy - 1 Jul 2018
Potter Rachael A, Griffin Danielle A, Sondergaard Patricia C, Johnson Ryan W, Pozsgai Eric R, Heller Kristin N, Peterson Ellyn L, Lehtimäki Kimmo K, Windish Hillarie P, Mittal Plavi J, Albrecht Douglas E, Mendell Jerry R, Rodino-Klapac Louise R
Abstract excerpt
Dysferlinopathies comprise a family of disorders caused by mutations in the dysferlin (DYSF) gene, leading to a progressive dystrophy characterized by chronic muscle fiber loss, fat replacement, and fibrosis. To correct the underlying histopathology and function, expression of full-length DYSF is required. Dual adeno-associated virus vectors have been developed, defined by a region of homology, to serve as a...
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