Cas9 nickase-mediated contraction of CAG/CTG repeats in vivo is accompanied by improvements in Huntington's disease pathology

Murillo Bartolome, Alvaro ORCID: https://orcid.org/0000-0001-5470-9347, Alpaugh, Melanie, Peter Durairaj, Ruban Rex, Randall, Emma L., Larin, Meghan, Heraty, Laura, Aston, Alys N., Taylor, Alysha S., Mas Monteys, Alex, Stoberl, Nina, Heuchan, Aeverie E. R., Aeschlimann, Pascale, Fears, Kyle, Landles, Christian, Osborne, Georgina F., Mangin, Antoine, Bhattacharyya, Soumyasree, Metzakopian, Emmanouil, Allen, Nicholas D. ORCID: https://orcid.org/0000-0003-4009-186X, Puymirat, Jack, Bates, Gillian P., Davidson, Beverly L., Cicchetti, Francesca, Lelos, Mariah J. ORCID: https://orcid.org/0000-0001-7102-055X and Dion, Vincent ORCID: https://orcid.org/0000-0003-4953-7637 2026. Cas9 nickase-mediated contraction of CAG/CTG repeats in vivo is accompanied by improvements in Huntington's disease pathology. Science Translational Medicine Item availability restricted.

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Item Type:	Article
Status:	In Press
Schools:	Schools > Medicine Schools > Biosciences
Publisher:	American Association for the Advancement of Science
ISSN:	1946-6234
Date of First Compliant Deposit:	15 October 2026
Date of Acceptance:	23 September 2026
Last Modified:	15 Oct 2026 09:00
URI:	https://orca.cardiff.ac.uk/id/eprint/190241

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