Dmd – animal models

Gene transfer therapy using systemic adeno-associated virus (AAV) delivery is being studied extensively for the treatment of monogenic diseases, such as Duchenne muscular dystrophy. Gene therapies are advancing rapidly, and it is crucial to optimize safety and efficacy as well as dosing of individuals with pre-existing antibodies against the vectors used for delivery. This nonhuman primate (NHP) study investigated the impact of various immunosuppression strategies on the safety and efficacy of gene transfer therapy and analyzed the safety and efficacy of therapeutic plasma exchange (TPE) as a potential pretreatment for individuals with pre-existing immunity.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research