In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

Over the past decade, vectors derived from adeno-associated virus (AAV) have established
themselves as a powerful tool for in vivo gene transfer, allowing long-lasting and safe …

Gene therapy mediated by recombinant adeno-associated virus (AAV) vectors is a
promising treatment for systemic monogenic diseases. However, vector immunogenicity …

Delivering genes to and across the brain vasculature efficiently and specifically across
species remains a critical challenge for addressing neurological diseases. We have evolved …

Neutralizing antibodies directed against adeno-associated virus (AAV) are commonly found
in humans. In seropositive subjects, vector administration is not feasible as antibodies …

Pompe disease (PD) is a rare autosomal recessive disorder caused by mutations in the GAA
gene, localized on chromosome 17 and encoding for acid alpha-1, 4-glucosidase (GAA) …

Glycogen storage disease type II or Pompe disease is a severe neuromuscular disorder
caused by mutations in the lysosomal enzyme, acid α-glucosidase (GAA), which result in …

Recombinant adeno-associated virus (AAV) vectors have been broadly adopted as a gene
delivery tool in clinical trials, owing to their high efficiency of transduction of several host …

Background & Aims Progressive familial intrahepatic cholestasis type 3 (PFIC3) is a rare
lethal autosomal recessive liver disorder caused by loss-of-function variations of the ABCB4 …

Hepatocyte-restricted, AAV-mediated gene transfer is being used to provide sustained,
tolerogenic transgene expression in gene therapy. However, given the episomal status of …