Work

504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy

Year: 2016

Type: article

Abstract: Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease characterized by progressive muscle wasting, loss of ambulation, and typically death in the third decade of life due to respiratory and cardiac complications. DMD results from deleterious mutations in the dystrophin gene that disrupts the translational reading frame and causes a complete lack of dystrophin protein. Becker muscular dystrophy (BMD) is similar to DMD in that it is the result of deletions in the dystrophin gene.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33313-5

Authors: Jacqueline N. Robinson-Hamm, Christopher E. Nelson, Ruth M. Castellanos Rivera, Annemieke Aartsma-Rus, Aravind Asokan, Charles A. Gersbach

Institutions: Duke University, University of North Carolina at Chapel Hill, Leiden University Medical Center

Language: English

Topic: CRISPR and Genetic Engineering

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,500