Department of Neurology, Institute of Neuroscience, Fujian Key Laboratory of Molecular Neurology, the First Affiliated Hospital, Fujian Medical University, 20 Chazhong Road, Fuzhou, 350005, China.
J Mol Med (Berl). 2022 Mar;100(3):385-394. doi: 10.1007/s00109-021-02167-y. Epub 2021 Nov 27.
Neurogenetic diseases are neurological conditions with a genetic cause (s). There are thousands of neurogenetic diseases, and most of them are incurable. The development of bioinformatics and elucidation of the mechanism of pathogenesis have allowed the development of gene therapy approaches, which show great potential in treating neurogenetic diseases. Viral vectors delivery, antisense oligonucleotides, gene editing, RNA interference, and burgeoning viroid delivery technique are promising gene therapy strategies, and commendable therapeutic effects in the treatment of neurogenetic diseases have been achieved (Fig. 1). This review highlights a sampling of advances in gene therapies for neurogenetic disorders. Fig. 1 Examples of gene therapy strategies used in the treatment of neurogenetic diseases. The schematic diagram shows different gene therapy approaches used for treating a sampling of neurogenetic disorders, such as ASO therapy, gene editing, gene augmentation, and RNA interference.
神经遗传病是由遗传原因引起的神经疾病（s）。有数千种神经遗传病，其中大多数是不可治愈的。生物信息学的发展和发病机制的阐明使基因治疗方法得以发展，这些方法在治疗神经遗传病方面显示出巨大的潜力。病毒载体传递、反义寡核苷酸、基因编辑、RNA 干扰和新兴的类病毒传递技术是很有前途的基因治疗策略，并在治疗神经遗传病方面取得了可喜的治疗效果（图 1）。本文综述了基因治疗神经遗传病方面的一些进展。图 1 用于治疗神经遗传病的基因治疗策略示例。该示意图显示了用于治疗神经遗传病的不同基因治疗方法，例如 ASO 治疗、基因编辑、基因增强和 RNA 干扰。
