Published in Science · View the paper (DOI)
Researchers have developed a CRISPR-Cas9 gene editing system to treat Duchenne muscular dystrophy (DMD), a debilitating genetic disease. By deleting exon 23 and restoring dystrophin protein levels, the therapy improved muscle function in mice with DMD, including cardiac and pulmonary health.
Coverage from 2 institutions
- Genetically correcting a muscle disorder American Association for the Advancement of Science (AAAS) · Dec 31, 2015 · first to report
- CRISPR treats genetic disorder in adult mammal Duke University · Dec 31, 2015