Splice modulators target PMS1 to reduce somatic expansion of the Huntington's disease-associated CAG repeat.
McLean ZL, Gao D, Correia K, Roy JCL, Shibata S, Farnum IN, Valdepenas-Mellor Z, Kovalenko M, Rapuru M, Morini E, Ruliera J, Gillis T, Lucente D, Kleinstiver BP, Lee JM, MacDonald ME, Wheeler VC, Mouro Pinto R, Gusella JF
Nat Commun. 2024 Apr 12;15(1):3182. doi: 10.1038/s41467-024-47485-0.
(Link opens in a new window)
PubMed
(Link opens in a new window)
Article
Plasmids from Article
No plasmids are currently publicly available from Addgene for this article.