Researchers are investigating the safety and effectiveness of a new gene therapy product that replaces the defective β-sarcoglycan gene.
Genome-wide association study identifies two variants that may yield therapies for children with DMD.
The model aims to provide the basis for many future studies to bring therapeutic options to patients with FSHD.
Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.
Recent applications of adeno-associated virus (AAV) mediated gene therapy have focused mainly on correcting recessively inherited diseases. But what about dominantly inherited disorders? It looks like AAV could be a delivery mechanism for treating those genetic disorders, too.
