Small molecule splicing modifiers with systemic HTT-lowering activity.
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Nat Commun. 2021 Dec 15;12(1):7299. doi: 10.1038/s41467-021-27157-z.
Nat Commun. 2021.
PMID: 34911927
Free PMC article.
Huntington's disease (HD) is a hereditary neurodegenerative disorder caused by expansion of cytosine-adenine-guanine (CAG) trinucleotide repeats in the huntingtin (HTT) gene. ...
Huntington's disease (HD) is a hereditary neurodegenerative disorder caused by expansion of cytosine-adenine-guanine (C …