New studies refocus attention on the genotoxicity of AAV vectors in gene therapy

(Mary Ann Liebert, Inc./Genetic Engineering News) A growing number of preclinical studies in mice suggests that therapeutic gene delivery using recombinant adeno-associated viral vectors (rAAVs) can cause insertional mutagenesis and increase the risk of hepatocellular carcinoma. Despite the apparent safety of rAAV-mediated gene therapy in human clinical applications, the data emerging from some mouse studies emphasize the need to carefully reconsider the potential risk of genotoxicity, according to the authors of a provocative article published in Human Gene Therapy.
Source: EurekAlert! - Medicine and Health - Category: Global & Universal Source Type: news