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

Alvaro Murillo; Melanie Alpaugh; Ruban Rex Peter Durairaj; Emma L Randall; Meghan Larin; Laura Heraty; Alys N Aston; Alysha S Taylor; Alex Mas Monteys; Nina Stöberl; Aeverie E R Heuchan; Pascale Aeschlimann; Kyle Fears; Christian Landles; Georgina F Osborne; Antoine Mangin; Soumyasree Bhattacharyya; Emmanouil Metzakopian; Nicholas D Allen; Jack Puymirat; Gillian P Bates; Beverly L Davidson; Francesca Cicchetti; Mariah J Lelos; Vincent Dion

doi:10.1126/scitranslmed.aef9466

SCHEDA SCIENTIFICA2026Altra ricerca

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

Alvaro Murillo, Melanie Alpaugh, Ruban Rex Peter Durairaj, Emma L Randall, Meghan Larin, Laura Heraty, Alys N Aston, Alysha S Taylor, Alex Mas Monteys, Nina Stöberl, Aeverie E R Heuchan, Pascale Aeschlimann, Kyle Fears, Christian Landles, Georgina F Osborne, Antoine Mangin, Soumyasree Bhattacharyya, Emmanouil Metzakopian, Nicholas D Allen, Jack Puymirat, Gillian P Bates, Beverly L Davidson, Francesca Cicchetti, Mariah J Lelos, Vincent Dion

Science translational medicine · 2026

Psicologia generale

Abstract e pubblicazione originale

L’abstract è consultabile nella fonte originale. Non viene ripubblicato qui perché non risulta verificata una licenza compatibile.

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Parole chiave

AnimalsHuntington DiseaseDisease Models, AnimalPhenotypeHumansTrinucleotide Repeat ExpansionDeoxyribonuclease IMiceNeuronsCRISPR-Cas SystemsMyotonic DystrophyAstrocytes

Come citare questo studio

Alvaro Murillo, Melanie Alpaugh, Ruban Rex Peter Durairaj, Emma L Randall, Meghan Larin, Laura Heraty, Alys N Aston, Alysha S Taylor, Alex Mas Monteys, Nina Stöberl, Aeverie E R Heuchan, Pascale Aeschlimann, Kyle Fears, Christian Landles, Georgina F Osborne, Antoine Mangin, Soumyasree Bhattacharyya, Emmanouil Metzakopian, Nicholas D Allen, Jack Puymirat, Gillian P Bates, Beverly L Davidson, Francesca Cicchetti, Mariah J Lelos, Vincent Dion. (2026). Cas9 nickase-mediated contraction of CAG/CTG repeats is associated with phenotypic improvements in a Huntington's disease mouse model.. Science translational medicine. DOI: 10.1126/scitranslmed.aef9466

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