New data highlights safety concerns on rAAV vectors used in gene therapy

New data highlight safety concerns for the replication of recombinant adeno-associated viral (rAAV) vectors commonly used in gene therapy. These findings, which emphasize the need for mobilization resistant AAV vectors, are reported in the peer-reviewed journal Human Gene Therapy.

The risk of rAAV mobilization has been underappreciated in the AAV research community.

The data generated herein highlight the potential of rAAV vector production in treated patients upon subsequent wtAAV and helper virus infection. They raise safety concerns for the treated individual and for the unintended animal and human populations in general."

Matthew Hirsch, PhD, Study Coauthor, University of North Carolina at Chapel Hill

"Viral gene therapy vectors are, by their nature, viruses capable of replicating as viruses under certain conditions. These studies help to define the conditions under which that might occur in patients receiving rAAV vectors," according to Editor-in-Chief of Human Gene Therapy Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School.