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

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 re...

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Bibliographic Details
Published in:Hum Gene Ther
Main Authors: 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.
Format: Artigo
Language:Inglês
Published: Mary Ann Liebert, Inc. 2018
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Research Articles
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6066196/
https://ncbi.nlm.nih.gov/pubmed/28707952
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.062
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