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Early whole-body mutant huntingtin lowering averts changes in proteins and lipids important for synapse function and white matter maintenance in the LacQ140 mouse model

2023-01-27

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> Expansion of a triplet repeat tract in exon1 of the HTT gene causes Huntington’s disease (HD). The mutant HTT protein (mHTT) has numerous aberrant interactions with diverse, pleiomorphic effects. No disease modifying treatments exist but lowering mutant huntingtin (mHTT) by gene therapy is a promising approach to treat Huntington’s disease (HD). It is not clear when lowering...

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Literature Corpus work
d69d794e-039f-5f97-bc50-b4e9b52b0734
DOI
10.1101/2023.01.26.525697
Open publication

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Early whole-body mutant huntingtin lowering averts changes in proteins and lipids important for synapse function and white matter maintenance in the LacQ140 mouse modelDOI 10.1101/2023.01.26.525697
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