An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.
Keller CG, Shin Y, Monteys AM, Renaud N, Beibel M, Teider N, Peters T, Faller T, St-Cyr S, Knehr J, Roma G, Reyes A, Hild M, Lukashev D, Theil D, Dales N, Cha JH, Borowsky B, Dolmetsch R, Davidson BL, Sivasankaran R.
Keller CG, et al. Among authors: borowsky b.
Nat Commun. 2022 Mar 3;13(1):1150. doi: 10.1038/s41467-022-28653-6.
Nat Commun. 2022.
PMID: 35241644
Free PMC article.