首页> eLife> Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease.

Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease.

导出 LinkOut

翻译打印收藏纠错

第一作者： Doo Eun,Choi

第一单位： Center for Genomic Medicine, Massachusetts General Hospital, Boston, United States.;Department of Neurology, Harvard Medical School, Boston, United States.

作者： Doo Eun,Choi ^[1] ; Jun Wan,Shin ^[1] ; Sophia,Zeng ^[2] ; Eun Pyo,Hong ^[3] ; Jae-Hyun,Jang ^[1] ; Jacob M,Loupe ^[1] ; Vanessa C,Wheeler ^[1] ; Hannah E,Stutzman ^[4] ; Ben,Kleinstiver ^[5] ; Jong-Min,Lee ^[3]

作者单位： Center for Genomic Medicine, Massachusetts General Hospital, Boston, United States.;Department of Neurology, Harvard Medical School, Boston, United States. ^[1] Center for Genomic Medicine, Massachusetts General Hospital, Boston, United States. ^[2] Center for Genomic Medicine, Massachusetts General Hospital, Boston, United States.;Department of Neurology, Harvard Medical School, Boston, United States.;Medical and Population Genetics Program, The Broad Institute of MIT and Harvard, Cambridge, United States. ^[3] Center for Genomic Medicine, Massachusetts General Hospital, Boston, United States.;Department of Pathology, Massachusetts General Hospital, Boston, United States. ^[4] Center for Genomic Medicine, Massachusetts General Hospital, Boston, United States.;Department of Pathology, Massachusetts General Hospital, Boston, United States.;Department of Pathology, Harvard Medical School, Boston, United States. ^[5]

关键词 CAG repeat instability CAG-to-CAA conversion Huntington's disease base editing duplicated interruption genetics genomics human uninterrupted repeat