Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, Worcester, Massachusetts, USA.
Department of Neurology, Worcester, Massachusetts, USA.
Hum Gene Ther. 2022 Sep;33(17-18):889-892. doi: 10.1089/hum.2022.170.
Adeno-associated virus (AAV)-mediated gene therapies have provided promising treatments for numerous neurological disorders. Redosing of AAV to the central nervous system (CNS) is an attractive research area due to both the somewhat immunologically privileged status of the CNS as well as the possibility of reduced glial transgene expression over time following a single injection. Continued study of the immune responses to both intraparenchymal and intra-CSF delivery of AAV mediated gene therapies, as well as the continued study of immunosuppressive regimens, could allow for eventual redosing in patients.
腺相关病毒（AAV）介导的基因治疗为许多神经疾病提供了有希望的治疗方法。由于中枢神经系统（CNS）具有一定的免疫特权地位，以及单次注射后神经胶质转基因表达随时间减少的可能性，因此对 CNS 中的 AAV 进行再给药是一个很有吸引力的研究领域。对脑实质内和脑室内 AAV 介导的基因治疗的免疫反应的持续研究，以及对免疫抑制方案的持续研究，可能允许在患者中进行最终的再给药。
