Risk of AAV mobilization 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 journalHuman Gene Therapy.
The risk of rAAV mobilization has been underappreciated in the AAV research community.
"The data generated herein highlight the potential of rAAVvectorproduction in treated patients upon subsequent wtAAV and helper virus infection," state Matthew Hirsch, Ph.D., University of North Carolina at Chapel Hill, and coauthors. "They raise safety concerns for the treated individual and for the unintended animal andhuman populationsin general."
"Viralgene therapyvectors 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," says editor in chief ofHuman Gene Therapy, Terence R. Flotte, MD.
更多的信息:Liujiang Song et al. Adeno-Associated Virus Vector Mobilization, Risk Versus Reality,Human Gene Therapy(2020).DOI: 10.1089/hum.2020.118
