Advances in gene therapy for neurogenetic diseases: a brief review

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.

Automated summary

Neurogenetic diseases are neurological conditions with a genetic cause that are mostly incurable. Gene therapy approaches such as viral vectors delivery, antisense oligonucleotides, and gene editing have shown promising therapeutic effects in treating neurogenetic diseases.