Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy
系统性递送 Dysferlin 重叠载体可实现 Dysferlin 病患者的长期基因表达和功能改善。
期刊:Human Gene Therapy
影响因子:4
doi:10.1089/hum.2017.062
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