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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Publicado en:Hum Gene Ther
Autores principales: 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.
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2018
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Research Articles
Acceso en línea: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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