Article
Huntington's disease phenotypes are improved via mTORC1 modulation by small molecule therapy.
PloS one - 1 Jan 2022
St-Cyr Sophie, Child Daniel D, Giaime Emilie, Smith Alicia R, Pascua Christine J, Hahm Seung, Saiah Eddine, Davidson Beverly L
Abstract excerpt
Huntington's Disease (HD) is a dominantly inherited neurodegenerative disease for which the major causes of mortality are neurodegeneration-associated aspiration pneumonia followed by cardiac failure. mTORC1 pathway perturbations are present in HD models and human tissues. Amelioration of mTORC1 deficits by genetic modulation improves disease phenotypes in HD models, is not a viable therapeutic strategy. Here, we...
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