Okada Takashi, Nomoto Tatsuya, Shimazaki Kuniko, Lijun Wang, Lu Yanyan, Matsushita Takashi, Mizukami Hiroaki, Urabe Masashi, Hanazono Yutaka, Kume Akihiro, Muramatsu Shin-ichi, Nakano Imaharu, Ozawa Keiya
Division of Genetic Therapeutics, Center for Molecular Medicine, Jichi Medical School, 3311-1 Yakushiji, Minami-Kawachi, Kawachi, Tochigi 329-0498, Japan.
Methods. 2002 Oct;28(2):237-47. doi: 10.1016/s1046-2023(02)00228-1.
Gene therapy is a novel method under investigation for the treatment of neurological disorders. Considerable interest has focused on the possibility of using viral vectors to deliver genes to the central nervous system. Adeno-associated virus (AAV) is a potentially useful gene transfer vehicle for neurologic gene therapies. The advantages of AAV vector include the lack of any associated disease with a wild-type virus, the ability to transduce nondividing cells, the possible integration of the gene into the host genome, and the long-term expression of transgenes. The development of novel therapeutic strategies for neurological disorder by using AAV vector has an increasing impact on gene therapy research. This article describes methods that can be used to generate rodent and nonhuman primate models for testing treatment strategies linked to pathophysiological events in the ischemic brain and neurodegenerative disorders such as Parkinson's disease.
基因治疗是一种正在研究中的用于治疗神经疾病的新方法。人们对使用病毒载体将基因传递到中枢神经系统的可能性给予了极大关注。腺相关病毒（AAV）是一种对神经基因治疗有潜在用途的基因传递载体。AAV载体的优点包括与野生型病毒无任何相关疾病、能够转导非分裂细胞、基因可能整合到宿主基因组以及转基因的长期表达。利用AAV载体开发神经疾病的新型治疗策略对基因治疗研究的影响日益增大。本文描述了可用于生成啮齿动物和非人类灵长类动物模型的方法，以测试与缺血性脑和帕金森病等神经退行性疾病中的病理生理事件相关的治疗策略。
