Advances in gene therapy for neurogenetic diseases: a brief review

• Published in: Journal of molecular medicine (Berlin, Germany) Vol. 100; no. 3; pp. 385 - 394
• Main Authors: Xie, Ying-Xuan, Lv, Wen-Qi, Chen, Yi-Kun, Hong, Shunyan, Yao, Xiang-Ping, Chen, Wan-Jin, Zhao, Miao
• Format: Journal Article
• Language: English
• Published: Berlin/Heidelberg Springer Berlin Heidelberg 01.03.2022; Springer Nature B.V
• Subjects: Antisense oligonucleotides; Antisense RNA; Bioinformatics; Biomedical and Life Sciences; Biomedicine; Expression vectors; Gene therapy; Genome editing; Human Genetics; Internal Medicine; Molecular Medicine; Review; RNA editing; RNA-mediated interference; Antisense oligonucleotides (ASOs); Gene therapy; Neurogenetic diseases; Gene editing
• ISSN: 0946-2716; 1432-1440; 1432-1440
• DOI: 10.1007/s00109-021-02167-y

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