dysferlin

Potter, R., Griffin, D., Sondergaard, P., Johnson, R., Pozsgai, E., Heller, K., … Rodino-Klapac, L. (2018). Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy. Human Gene Therapy, 29(7), 749-762. https://doi.org/10.1089/hum.2017.062 (Original work published 2018)