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.
By removing a barrier to access – production of pharmaceutical products for clinical trial – the team at the GMP facility is bringing more treatments to rare diseases.
A first-of-its-kind oligodendrocyte in vitro model shows that human cells normally supportive of motor neuron function play an active role in amyotrophic lateral sclerosis pathogenesis – and this discovery may point the way toward therapeutic timing and targets.
