Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

Abstract Background Patients with the Crigler–Najjar syndrome lack the enzyme uridine
diphosphoglucuronate glucuronosyltransferase 1A1 (UGT1A1), the absence of which leads …

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 …

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

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 is poised to revolutionize modern medicine, with seemingly unlimited potential
for treating and curing genetic disorders. For otherwise incurable indications, including most …

Pre-existing immunity to adeno-associated virus (AAV) is highly prevalent in humans and
can profoundly impact transduction efficiency. Despite the relevance to AAV-mediated gene …

Glycogen storage disease type III (GSDIII) is a rare inborn error of metabolism affecting liver,
skeletal muscle, and heart due to mutations of the AGL gene encoding for the glycogen …

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 …