Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for ...

Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...

Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene ...

People with Duchenne muscular dystrophy carry a mutation in the DMD gene coding for dystrophin, a protein crucial for proper muscle function. Scientists are working to treat the disease at its genetic ...

Duchenne muscular dystrophy (DMD), the most lethal musculoskeletal disease, is caused by mutations in the X-linked gene dystrophin. Patients with DMD show delayed motor milestone development at 2-6 ...

Researchers report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne ...

UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section ...

Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop ...

Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. On August 24 in the journal Stem Cell Reports, researchers show how a dual CRISPR ...