Recent Advances in the Treatment of Huntington's Disease: Targeting DNA and RNA

被引：25

作者：

Shannon, Kathleen M. ^{[1
]}

机构：

[1] Univ Wisconsin Sch Med, Dept Neurol, Publ Hlth, 1685 Highland Ave #7158, Madison, WI 53705 USA

来源：

CNS DRUGS | 2020年 / 34卷 / 03期

关键词：

SINGLE-NUCLEOTIDE POLYMORPHISMS; MUTANT HUNTINGTIN; ANTISENSE OLIGONUCLEOTIDES; MOLECULAR PATHOGENESIS; MEDIATED DELIVERY; CLINICAL-TRIALS; MOUSE MODEL; WEIGHT-LOSS; IN-VITRO; THERAPY;

D O I：

10.1007/s40263-019-00695-3

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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.

引用

页码：219 / 228

页数：10

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