Biology: Risk of AAV mobilization in gene therapy

New Rochelle, NY, November 11, 2020–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. Click here to read the full-text article free through December 11, 2020.

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,” state Matthew Hirsch, PhD, University of North Carolina at Chapel Hill, and coauthors. “They raise safety concerns for the treated individual and for the unintended animal and human populations in general.”

“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.

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Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available information and a sample issue, please visit the Human Gene Therapy website.

Mary Ann Liebert, Inc., publishers is known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s 90 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.