Gene therapy with adeno-associated viral (AAV) vectors has reached the clinical stage for
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …

Clinical gene therapy has been increasingly successful owing both to an enhanced
molecular understanding of human disease and to progressively improving gene delivery …

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 …

Recombinant adeno-associated virus vectors (rAAV) are being explored as gene delivery
vehicles for the treatment of various inherited and acquired disorders. rAAVs are attractive …

Research over the past 20 years has focused on the development of adenoassociated virus
(AAV) DNA delivery vectors for the treatment of several hereditary human diseases. Among …

Abstract Adeno-associated virus (AAV)-mediated gene therapy has evolved from bench to
bedside, and now is the therapy of choice for certain inherited diseases. However, the small …

Gene therapy remains an attractive form of treatment for a variety of diseases, both inherited
and acquired. Recent experience in clinical gene therapy has highlighted important safety …

Gene therapy vectors based on the adeno-associated virus (AAV) are being developed for a
widening variety of therapeutic applications. Enthusiasm for AAV is due, not only to the …

Adeno-associated virus (AAV) derived vectors are considered highly eligible vehicles for
human gene therapy. Not only do they possess many great potential for clinical applications …

Gene delivery vectors based on adeno-associated virus (AAV) have been utilized in a large
number of gene therapy clinical trials, which have demonstrated their strong safety profile …