Article

A novel imaging ligand as a biomarker for mutant huntingtin-lowering in Huntington’s disease

2021-07-11

Abstract excerpt

Huntington’s disease (HD) is a dominantly inherited neurodegenerative disorder caused by a CAG trinucleotide expansion in the huntingtin ( HTT ) gene that encodes the pathologic mutant HTT (mHTT) protein with an expanded polyglutamine (PolyQ) tract. While several therapeutic programs targeting mHTT expression have advanced to clinical evaluation, no method is currently available to visualize mHTT levels in the l...

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Literature Corpus work: 5e4b4263-a11b-5b28-a35d-8cb529f8f916
DOI: 10.1101/2021.07.09.451725

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