Huntington's disease is a dominantly inherited neurodegenerative disease caused by an unstable expanded trinucleotide repeat at the short end of the fourth chromosome. Central nervous system pathology begins in the striatum, eventually affecting the entire brain and occurs consequent to multiple intracellular derangements. The proximate cause is a mutant protein with an elongated polyglutamine tract. Pharmacological approaches targeting multiple domains of intracellular functions have universally been disappointing. However, recent developments in gene therapy, including antisense oligonucleotides, small interfering RNAs, and gene editing are bringing new hope to the Huntington's community. This review discusses the promises and challenges of these new potential treatments.

中文翻译：

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亨廷顿氏病治疗的最新进展：靶向DNA和RNA。

亨廷顿舞蹈病是由遗传性遗传性遗传遗传疾病，由第四条染色体的短端不稳定的扩展三核苷酸重复序列引起。中枢神经系统病理始于纹状体，最终影响整个大脑，并因细胞内多种紊乱而发生。最直接的原因是具有延长的聚谷氨酰胺束的突变蛋白。靶向细胞内功能的多个结构域的药理方法普遍令人失望。但是，包括反义寡核苷酸，小干扰RNA和基因编辑在内的基因疗法的最新发展为亨廷顿氏族带来了新希望。这篇综述讨论了这些新的潜在疗法的前景和挑战。