AAV Vectors, Insertional Mutagenesis, and Cancer

Abstract

By now we have all heard a lot about retroviral vectors and cancer, but other types of vectors have typically been left out of these discussions. However, results presented at the recent American Society of Gene Therapy meeting and just published in Science1 show that this is a subject that goes beyond retroviruses, hematopoietic cells, and gamma C transgenes. In this study, mice developed hepatocellular carcinomas (HCCs) that contained adeno-associated virus (AAV) vector proviruses at a specific chromosomal locus, raising important questions about the safety of in vivo gene therapy.