Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Year: 2018
Type: article
Abstract: Gene editing and muscular dystrophy Duchenne muscular dystrophy (DMD) is characterized by progressive muscle weakness and a shortened life span. The disease is caused by mutations that reduce or prevent expression of dystrophin, an essential structural protein in skeletal and heart muscle. The gene editing technology CRISPR-Cas9 can correct disease-causing mutations and has yielded promising results in mouse models of DMD. In a small, short-term study, Amoasii et al. tested this strategy in a dog model of DMD that exhibits many features of the human disease. Intramuscular or systemic delivery of the gene editing components resulted in a substantial increase in dystrophin protein levels in skeletal and heart muscle. Restoration of dystrophin expression was accompanied by improved muscle histology. Science , this issue p. 86
Source: Science
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: green
Funders: Welch Foundation, Wellcome Trust, National Institutes of Health, National Heart, Lung, and Blood Institute, National Institute of Diabetes and Digestive and Kidney Diseases, National Institute of Arthritis and Musculoskeletal and Skin Diseases, Eunice Kennedy Shriver National Institute of Child Health and Human Development
Awards: Heart Repair through Reprogramming of Cardiac Cell Fate, Developing and phenotyping a colony of dogs with dystrophin-deficient muscular dystrophy: a strategic resource for UK-based research into Duchenne Muscular Dystrophy., G662211534, Molecular Dissection of Myoblast Fusion In Muscle Development and Regeneration, Deciphering the role of a novel micropeptide in cardiac function and dysfunction, Metabolic Control by Myogenic MicroRNAs, Mediator and Myokines, Myoediting of Duchenne muscular dystrophy
OpenAlex ID: W2889510460