Advancements in gene therapy for Huntington's disease

Abstract:

Huntington's disease is an autosomal dominant genetic disease. In recent years，clinical trials have been conducted for various intervention strategies targeting mRNA levels，and meanwhile，with the development of the clustered regularly interspersed short palindromic repeat（CRISPR）/CRISPR-associated genes system，there are also reports on gene editing strategies for pathogenic genomes. This article reviews the gene therapy for Huntington's disease in terms of current clinical status，research advances，and improvements in clinical assessment.