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Updated: Jun 27, 2026

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高效类治疗药物 防止在亨廷顿病中蛋白质聚合

Anooshay Khan1,2, Cemile Elif Özçelik1, Ozge Begli1

  • 1UNAM-Institute of Materials Science and Nanotechnology, Bilkent University, 06800 Ankara, Turkey.

ACS medicinal chemistry letters
|December 20, 2023
PubMed
概括

亨廷顿病 (HD) 疗法通过新的类药物得到了推进. 这些抑制了与突变的亨廷丁 (mHtt) 蛋白相关的有毒蛋白聚合物的形成,提供了有前途的治疗.

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Huntington Disease l: Introduction01:21

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Huntington disease or HD is a progressive, fatal neurodegenerative disorder inherited in an autosomal dominant pattern.PathophysiologyIt is caused by expansion of the CAG trinucleotide repeat in the HTT gene on chromosome 4 (4p16.3), producing an abnormal huntingtin protein with an expanded polyglutamine tract. This misfolded protein disrupts cellular function, leading to neuronal death. Normal alleles have ≤26 repeats, 27–35 are intermediate (risk of expansion), 36–39 show reduced penetrance,...