Article

Global Huntingtin Knockout in Adult Mice Leads to Fatal Neurodegeneration that Spares the Pancreas

2024-01-12

Abstract excerpt

Huntington’s disease (HD) is a fatal neurogenerative disorder caused by an expanded glutamine-coding CAG tract in the Huntingtin (Htt) gene. HD is believed to primarily arise via a toxic gain of function, and as a result a wide range of Htt-lowering treatments are in clinical trials. The safety of these trials is contingent on the risks imposed by Htt lowering: Htt is widely conserved, ubiquitously expressed and i...

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Literature Corpus work: dbee1914-c8a9-5425-94a8-eb97bca60c76
DOI: 10.1101/2024.01.11.575238

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