Emerging therapeutic approaches of neurogenetic diseases / 中华神经科杂志

ABSTRACT

Neurogenetic diseases are a group of hereditary diseases that predominantly affect the nervous system, including the central nervous system, peripheral nerves, and muscles. The clinical manifestations are complex, and the treatments are rather difficult. In recent years, with the in-depth study of the etiology and pathogenesis of neurogenetic diseases, gene therapeutic approaches developed for mutant genes, such as antisense oligonucleotides, RNA interference, adeno-associated virus-mediated gene transduction, small molecule compounds with gene modification effects, and clustered regularly interspaced short palindromic repeats/Cas9 gene editing strategies are gradually transformed into clinical applications. The drugs with potential therapeutic effects are developed in according to the promising targets within the pathogenic mechanism of diseases. These emerging therapeutic approaches provide innovative prospects for patients with neurogenetic diseases.