Abstract:Huntington's disease is an autosomal dominant hereditary disease. Gene therapy is an effective means to control the progression of the disease and even to achieve radical cure. With the initial success of antisense oligonucleotide therapy for Huntington's disease, different strategies targeting genomic DNA or mRNA are being actively developed and improved, and clinical trials will be carried out in the near future. This review focuses on the current situation, further development, and clinical challenges of gene therapy for Huntington's disease.
