亨廷顿病基因治疗的进展与挑战

Progress and challenges in gene therapy for Huntington's disease

摘要:

亨廷顿病是一种常染色体显性遗传病，基因治疗是控制病情进展甚至实现根治的有效手段。随着反义核苷酸疗法在亨廷顿病基因治疗中获得初步成功，针对基因组水平及mRNA水平的各项技术也正在积极开发、改良，在不久的将来相继开展临床试验。本文围绕亨廷顿病基因治疗的现状、发展方向及临床挑战，开展相关研究状况的综述。

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.