Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy-Reference-Cited by-同舟云学术

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

Published:2018-07 Issue:7 Volume:29 Page:749-762
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Potter Rachael A.¹,Griffin Danielle A.¹,Sondergaard Patricia C.¹,Johnson Ryan W.¹,Pozsgai Eric R.¹²,Heller Kristin N.¹,Peterson Ellyn L.¹,Lehtimäki Kimmo K.³,Windish Hillarie P.⁴,Mittal Plavi J.⁴,Albrecht Douglas E.⁴,Mendell Jerry R.¹⁵,Rodino-Klapac Louise R.¹⁵²

Affiliation:

1. Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.

2. Integrated Biomedical Science Graduate Program, College of Medicine, The Ohio State University, Columbus, Ohio; The Ohio State University, Columbus, Ohio.

3. Charles River Discovery Services, Kuopio, Finland.

4. The Jain Foundation, Seattle, Washington.

5. Department of Pediatrics and Neurology, The Ohio State University, Columbus, Ohio; The Ohio State University, Columbus, Ohio.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2017.062

Reference48 articles.

1. Defective membrane repair in dysferlin-deficient muscular dystrophy

2. Limb-Girdle Muscular Dystrophy in the United States