■特集〔遺伝子治療update：日本医科大学の遺伝子治療研究（4）〕
アデノ随伴ウイルス（AAV）ベクターの感染細胞へのゲノム組み込みについて
平井 幸彦
東京大学医科学研究所遺伝子・細胞治療センター分子遺伝学分野
Genome Integration of Adeno-associated Virus (AAV) Vectors into Infected Cells
Yukihiko Hirai
Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo
The development of gene therapy products using adeno-associated virus (AAV) vectors is progressing, and the gene therapy market is rapidly expanding. AAV shows no pathogenicity in the human body, has extremely low cytotoxicity and, unlike lentiviral and retroviral vectors, rarely integrates into chromosomes. Consequently, risk associated with AAV was thought to be low. In recent years, however, adverse events such as hepatotoxicity have become apparent as cases accumulate among laboratory animals and in clinical trials. In this paper, we present an overview of the integration of AAV vectors into the genome of infected cells, which is thought to be the cause of adverse events.
日医大医会誌 2023; 19(3), 211-217
Key words
adeno-associted virus vector (rAAV), integration into genomic DNA, insertional mutagenesis, the risk of rAAV-mediated oncogenesis
Correspondence to
Yukihiko Hirai, Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo 4-6-1 Shirokanedai, Minato-ku,Tokyo 108-8639, Japan
E-mail：hirai@ims.u-tokyo.ac.jp
受付：2023年6月26日 受理：2023年7月3日 |
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