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Peer-reviewedLicence not statedScience Translational Medicine

Cas9 nickase–mediated contraction of CAG/CTG repeats is associated with phenotypic improvements in a Huntington’s disease mouse model

  • Alvaro Murillo1
  • Melanie Alpaugh2
  • Ruban Rex Peter Durairaj1
  • Emma L. Randall1
  • Meghan Larin3,1
  • Laura Heraty1
  • Alys N Aston1
  • Alysha S Taylor1
  • Alex Mas Monteys4
  • Nina Stöberl5
  • Aeverie E. R. Heuchan1
  • Pascale C. Aeschlimann1
13 more authors
  • Kyle Fears
  • Christian Landles
  • Georgina F Osborne
  • Antoine Mangin
  • Soumyasree Bhattacharyya1
  • Emmanouil Metzakopian
  • Nicholas D. Allen5
  • Jack Puymirat6
  • Gillian P. Bates
  • Beverly L. Davidson4
  • Francesca Cicchetti7
  • Mariah J. Lelos5
  • Vincent Dion8,1
8 affiliations
  1. 1UK Dementia Research Institute at Cardiff University, Hadyn Ellis Building, Maindy Road, Cardiff, United Kingdom, CF24 4HQ
  2. 2Department of Molecular and Cellular Biology, University of Guelph, Guelph, Ontario, Canada N1G 2W1
  3. 3AstraZeneca R&D 1 Francis Crick Avenue Cambridge, UK CB2 0AA
  4. 4Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia, Philadelphia, 19104, USA
  5. 5School of Biosciences, Cardiff University, The Sir Martin Evans Building, Museum Avenue, Cardiff, CF10 3AX
  6. 6LOEX, CHU de Québec-Université Laval Research Center, Quebec City, QC, Canada
  7. 7Centre de Recherche du CHU de Québec, Axe Neurosciences; Département de Psychiatrie et Neurosciences, Université Laval, Québec, QC, Canada
  8. 8Division of Psychological Medicine and Clinical Neurosciences, Cardiff University, Maindy Road, Cardiff, UK, CF24 4HQ
Open at sourceDOICite

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Cite

BibTeXRIS
BibTeX
@article{murillo2026cas9,
  title = {{Cas9 nickase–mediated contraction of CAG/CTG repeats is associated with phenotypic improvements in a Huntington’s disease mouse model}},
  author = {Alvaro Murillo and Melanie Alpaugh and Ruban Rex Peter Durairaj and Emma L. Randall and Meghan Larin and Laura Heraty and Alys N Aston and Alysha S Taylor and Alex Mas Monteys and Nina Stöberl and Aeverie E. R. Heuchan and Pascale C. Aeschlimann and Kyle Fears and Christian Landles and Georgina F Osborne and Antoine Mangin and Soumyasree Bhattacharyya and Emmanouil Metzakopian and Nicholas D. Allen and Jack Puymirat and Gillian P. Bates and Beverly L. Davidson and Francesca Cicchetti and Mariah J. Lelos and Vincent Dion},
  journal = {Science Translational Medicine},
  year = {2026},
  month = oct,
  doi = {10.1126/scitranslmed.aef9466},
  url = {https://doi.org/10.1101/2024.02.19.580669},
}
RIS
TY  - JOUR
TI  - Cas9 nickase–mediated contraction of CAG/CTG repeats is associated with phenotypic improvements in a Huntington’s disease mouse model
AU  - Alvaro Murillo
AU  - Melanie Alpaugh
AU  - Ruban Rex Peter Durairaj
AU  - Emma L. Randall
AU  - Meghan Larin
AU  - Laura Heraty
AU  - Alys N Aston
AU  - Alysha S Taylor
AU  - Alex Mas Monteys
AU  - Nina Stöberl
AU  - Aeverie E. R. Heuchan
AU  - Pascale C. Aeschlimann
AU  - Kyle Fears
AU  - Christian Landles
AU  - Georgina F Osborne
AU  - Antoine Mangin
AU  - Soumyasree Bhattacharyya
AU  - Emmanouil Metzakopian
AU  - Nicholas D. Allen
AU  - Jack Puymirat
AU  - Gillian P. Bates
AU  - Beverly L. Davidson
AU  - Francesca Cicchetti
AU  - Mariah J. Lelos
AU  - Vincent Dion
PY  - 2026
DA  - 2026/10/21
JO  - Science Translational Medicine
DO  - 10.1126/scitranslmed.aef9466
UR  - https://doi.org/10.1101/2024.02.19.580669
SN  - 1946-6234
SN  - 1946-6242
ER  -