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.
The phase 1 clinical study shows that gene therapy extends survival of patients and supports achievement of milestones previously unseen in the natural course of the disease.
Louise Rodino-Klapac, PhD, principal investigator in the Center for Gene Therapy, shares an update on the advancement of LGMD2E gene therapy to clinical studies.
