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通过分子 tweezer CLR01 抑制亨廷丁外子-1 聚合

Tobias Vöpel1, Kenny Bravo-Rodriguez2, Sumit Mittal2

  • 1Department of Physical Chemistry II, Ruhr-University Bochum , 44780 Bochum, Germany.

Journal of the American Chemical Society
|April 14, 2017
PubMed
概括

亨廷顿蛋白 (htt) 的N端片段 (N17) 是亨廷顿病聚合的关键. 分子针CLR01向N17,抑制了HT的聚合和毒性.

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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,...