Science· 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·· 16 小时前评分61
Cas9 nickase介导的CAG/CTG重复序列收缩与亨廷顿病小鼠模型表型改善相关
Cas9 nickase–mediated contraction of CAG/CTG repeats is associated with phenotypic improvements in a Huntington’s disease mouse model
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Cas9 nickase介导的CAG/CTG重复序列收缩与亨廷顿病小鼠模型的表型改善相关。文章发表于Science Translational Medicine第18卷第872期（2026年10月），当前材料未提供完整正文、样本量或具体效应量。
来源：Science