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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Shranjeno v:
Bibliografske podrobnosti
izdano v: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
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc. 2018
Teme:
Research Articles
Online dostop: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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