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In vivo CRISPR–Cas9 genome editing in mice identifies genetic modifiers of somatic CAG repeat instability in Huntington’s disease

Crossref DOI link: https://doi.org/10.1038/s41588-024-02054-5

Published Online: 2025-01-22

Published Print: 2025-02

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Mouro Pinto, Ricardo https://orcid.org/0000-0001-6744-2805
Murtha, Ryan

Azevedo, António

Douglas, Cameron https://orcid.org/0000-0003-2334-1652
Kovalenko, Marina

Ulloa, Jessica

Crescenti, Steven

Burch, Zoe

Oliver, Esaria

Kesavan, Maheswaran

Shibata, Shota https://orcid.org/0000-0002-9131-5408
Vitalo, Antonia

Mota-Silva, Eduarda

Riggs, Marion J. https://orcid.org/0000-0002-7310-1107
Correia, Kevin

Elezi, Emanuela

Demelo, Brigitte

Carroll, Jeffrey B.

Gillis, Tammy

Gusella, James F. https://orcid.org/0000-0003-0681-9263
MacDonald, Marcy E.

Wheeler, Vanessa C. https://orcid.org/0009-0004-8259-5796
Funding

Funding for this research was provided by:

U.S. Department of Health & Human Services | NIH | Office of Extramural Research, National Institutes of Health (NS049206, NS111066, NS049206, NS126420, NS049206, NS049206, NS049206, NS049206, NS049206, NS049206, NS111066, NS091161, NS091161, NS049206, NS126420)

CHDI Foundation

Pfizer

Huntington’s Disease Society of America

Harvard NeuroDiscovery Center

National Ataxia Foundation

U.S. Department of Health & Human Services | National Institutes of Health (NS049206)

Dake Family Foundation
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Text and Data Mining valid from 2025-01-22

Version of Record valid from 2025-01-22
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Article History

Received: 24 May 2024

Accepted: 6 December 2024

First Online: 22 January 2025

Competing interests

: R.M.P. and V.C.W. received research support from Pfizer for this study. J.F.G. and V.C.W. were founding scientific advisory board members with a financial interest in Triplet Therapeutics. Their financial interests were reviewed and are managed by MGH and Mass General Brigham (MGB) in accordance with their conflict-of-interest policies. V.C.W. is a scientific advisory board member of LoQus23 Therapeutics and has provided paid consulting services to Acadia Pharmaceuticals, Alnylam, Biogen, Passage Bio and Rgenta Therapeutics. J.F.G. consults for Transine Therapeutics (dba Harness Therapeutics) and has previously provided paid consulting services to Wave Therapeutics USA, Biogen and Pfizer. The other authors declare no competing interests.

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