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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書誌詳細
出版年:Hum Gene Ther
主要な著者: 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.
フォーマット: Artigo
言語:Inglês
出版事項: Mary Ann Liebert, Inc. 2018
主題:
Research Articles
オンライン･アクセス: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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