Article

Quantifying mutant huntingtin protein in human cerebrospinal fluid to support the development of huntingtin-lowering therapies

2022-07-22

Abstract excerpt

<h4>Background</h4> Huntington’s disease (HD) is caused by a cytosine adenine guanine-repeat expansion in the huntingtin gene. This results in the production of toxic mutant huntingtin protein (mHTT), which has an elongated polyglutamine (polyQ) stretch near the protein’s N-terminal end. The pharmacological lowering of mHTT expression in the brain targets the underlying driver of HD and is one of the principal the...

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Literature Corpus work: e0158b59-0ec3-5372-ae1b-95ba437a160f
DOI: 10.1101/2022.07.22.22277927

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