Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions.

ABSTRACT: In the last two decades, recombinant adeno-associated virus has emerged as the most popular gene therapy vector. Recently AAV gene therapy has been approved by the FDA for the treatment of two rare genetic disorders, namely the early childhood blindness disease Leber congenital amaurosis and spinal muscular atrophy (SMA). As is the case for the treatment of SMA, if the AAV vector must be administered systemically, very high vector doses are often required for therapeutic efficacy. But higher vector doses inevitably increase the risk of adverse events. The tragic death of three children in a clinical trial to treat X-linked myotubular myopathy with an AAV vector has thrown this limitation into sharp relief. Regardless of the precise cause(s) that led to the death of the two children, it is

SUBMITTER: Riyad JM 

PROVIDER: S-EPMC8413391 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature