![Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Functional Improvement for Dysferlinopathy]()
Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Functional Improvement for DysferlinopathyPotter, 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. · Human Gene Therapy · 2017