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

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...