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亨廷顿舞蹈症:治疗的新希望

Huntington's disease: new hope for therapeutics.

作者信息

McMurray C T

机构信息

Dept Molecular Pharmacology and Experimental Therapeutics, Mayo Clinic and Foundation, Rochester, MN 55905, USA.

出版信息

Trends Neurosci. 2001 Nov;24(11 Suppl):S32-8. doi: 10.1016/s0166-2236(00)01997-4.

Abstract

Huntington's disease (HD) is one of eight progressive neurodegenerative disorders in which the underlying mutation is a CAG expansion encoding a polyglutamine tract. There are currently no cures or even effective therapies for HD. Effective strategies have remained elusive because little is known about either the mechanisms of expansion or the mechanism of polyglutamine-mediated neuronal death. However, recent advances in understanding the basic mechanisms of expansion and toxicity have renewed hope that a therapeutic strategy might someday be possible. Strategies effective in the treatment of HD are likely to be relevant in the treatment of a range of neurological and neurodegenerative disorders.

摘要

亨廷顿舞蹈症(HD)是八种进行性神经退行性疾病之一,其潜在突变是编码多聚谷氨酰胺序列的CAG重复扩增。目前尚无治愈HD的方法,甚至没有有效的治疗手段。由于对CAG重复扩增机制以及多聚谷氨酰胺介导的神经元死亡机制了解甚少,有效的治疗策略一直难以实现。然而,最近在理解扩增和毒性基本机制方面取得的进展,重新燃起了人们对未来某天可能找到治疗策略的希望。对HD有效的治疗策略可能也适用于一系列神经和神经退行性疾病的治疗。

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