Gene therapy products for the treatment of genetic diseases are currently in clinical trials,
and one of these, an adeno-associated viral (AAV) product, has recently been licensed. AAV …

Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

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 …

One of the major goals of in vivo gene transfer is to achieve long-term expression of
therapeutic transgenes in terminally differentiated cells. The extensive clinical experience …

Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …

Gene transfer trials with adeno-associated virus (AAV) vectors have initiated to unveil the
therapeutic potential of this approach, with some of the most exciting results coming from …

Findings in the first clinical trial in which an adeno-associated virus (AAV) vector was
introduced into the liver of human subjects highlighted an issue not previously identified in …

Recent findings in a clinical trial in which an adeno-associated virus (AAV) vector
expressing coagulation factor IX (F. IX) was introduced into the liver of hemophilia B subjects …

Original reports of adeno-associated virus (AAV) vector-mediated gene transfer to the
muscle resulted in high-level β-galactosidase (β-gal) expression and the promise of a viral …

Another confounding factor is the presence of other types of vector-related impurities
generated in the biosynthetic milieu of vector production, albeit at much lower levels. For …