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

Potter, Rachael A.; Griffin, Danielle A.; Sondergaard, Patricia C.; Johnson, Ryan W.; Pozsgai, Eric R.; Heller, Kristin N; Peterson, Ellyn L.; Lehtimaki, Kimmo K.; Windish, Hillarie P.; Mittal, Plavi J.; Albrecht, Doug E.; Mendell, Jerry R.; Rodino-Klapac, Louise R.

期刊名称Human Gene Therapy
作者Potter, Rachael A.; Griffin, Danielle A.; Sondergaard, Patricia C.; Johnson, Ryan W.; Pozsgai, Eric R.; Heller, Kristin N; Peterson, Ellyn L.; Lehtimaki, Kimmo K.; Windish, Hillarie P.; Mittal, Plavi J.; Albrecht, Doug E.; Mendell, Jerry R.; Rodino-Klapac, Louise R.
印刷ISSN1043-0342
电子ISSN1557-7422
文件大小1460.65KB
发表时间2017-07-14
DOI10.1089/hum.2017.062

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