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Intrastriatal Delivery of a Zinc Finger Protein Targeting the Mutant HTT Gene Allele Obviates Lipid Phenotypes in Brain and Plasma in Huntington's Disease Mice.

Human gene therapy - 1 Aug 2025

Iwanowicz Andrew, Boudi Adel, Seeley Connor, Sapp Ellen, Miller Rachael, Liu Sophia, Chase Kathryn, Shing Kai, Batista Ana Rita, Siena-Esteves Miguel, Aronin Neil, DiFiglia Marian, Kegel-Gleason Kimberly B

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...

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PMID: 40711410
DOI: 10.1177/10430342251359955

Genes

HTT

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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