Department of Medical Genetics and Pediatrics, 7 Chung-Shan S. Road, National Taiwan University Hospital, Taipei, Taiwan.
Division of Neurological Gene Therapy, Center for Open Innovation, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan.
Curr Gene Ther. 2022;22(3):185-190. doi: 10.2174/1566523221666210922155413.
Pre existing immunity to adeno-associated virus (AAV) poses a concern in AAV vector- mediated gene therapy. Localized administration of low doses of carefully chosen AAV serotypes can mitigate the risk of an immune response. This article will illustrate the low risk of immune response to AAV serotype 2 vector-mediated gene therapy to the brain with support from clinical trial data in aromatic L-amino acid decarboxylase deficiency and Parkinson disease.
腺相关病毒（AAV）预先存在的免疫性是 AAV 载体介导的基因治疗中的一个关注点。局部给予低剂量精心选择的 AAV 血清型可以降低免疫反应的风险。本文将通过芳香族 L-氨基酸脱羧酶缺乏症和帕金森病的临床试验数据支持，说明脑内 AAV 血清型 2 载体介导的基因治疗引起免疫反应的风险低。
