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An alternative splicing modulator decreases mutant HTT and improves the molecular fingerprint in Huntington's disease patient neurons.
Nat Commun. 2022 Nov 10;13(1):6797. doi: 10.1038/s41467-022-34419-x.
Nat Commun. 2022.
PMID: 36357392
Free PMC article.
Clinical Trial.
Suppression of Huntington's Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding.
Mathews EW, Coffey SR, Gärtner A, Belgrad J, Bragg RM, O'Reilly D, Cantle JP, McHugh C, Summers A, Fentz J, Schwagarus T, Cornelius A, Lingos I, Burch Z, Kovalenko M, Andrew MA, Frank Bennett C, Kordasiewicz HB, Marchionini DM, Wilkinson H, Vogt TF, Pinto RM, Khvorova A, Howland D, Wheeler VC, Carroll JB.
Mathews EW, et al. Among authors: lingos i.
bioRxiv [Preprint]. 2024 Nov 4:2024.11.04.619693. doi: 10.1101/2024.11.04.619693.
bioRxiv. 2024.
PMID: 39574582
Free PMC article.
Preprint.
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