Article

<i>HTT</i> silencing delays onset and slows progression of Huntington’s disease like phenotype: Monitoring with a novel neurovascular biomarker

2020-11-19

Abstract excerpt

Huntington’s disease (HD) is a dominantly inherited, fatal neurodegenerative disorder caused by a CAG expansion in the Huntingtin ( HTT ) gene, coding for pathologic mutant HTT protein (mHTT). Because of its gain-of-function mechanism and monogenic etiology, strategies to lower HTT are being actively investigated as disease-modifying therapies. Most approaches are currently targeted at the manifest HD stage, whe...

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Literature Corpus work: 390226f9-a950-5175-8208-a52ef2713883
DOI: 10.1101/2020.11.17.386631

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