Article

Intrastriatal delivery of a zinc finger protein targeting the mutant HTT gene by AAV9 obviates lipid phenotypes in brain and plasma of zQ175DN HD mice

2025-01-07

Abstract excerpt

Reducing the burden of mutant Huntingtin (mHTT) protein in brain cells is a strategy for treating Huntington’s disease (HD). However, it is still unclear what pathological changes can be reproducibly reversed by mHTT lowering and whether these changes can be measured in peripheral biofluids. We previously found that lipid changes that occur in brain with HD progression could be prevented by attenuating HTT transcr...

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Literature Corpus work: 69e07b6f-2329-5ea8-88d8-cb501a3e9c72
DOI: 10.1101/2025.01.06.630918

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