rAAV-CRISPRa therapy corrects Rai1 haploinsufficiency and rescues selective disease features in Smith-Magenis syndrome mice.

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作者： Hao-Cheng,Chang ^[1] ; Yu-Ju,Lee ^[1] ; Sehrish,Javed ^[1] ; Minza,Haque ^[1] ; Ya-Ting,Chang ^[1] ; Yu Cheng,Lin ^[1] ; Cameron,Oram ^[1] ; Wei-Hsiang,Huang ^[2]

作者单位： Department of Neurology and Neurosurgery, Centre for Research in Neuroscience, McGill University, Québec, Canada; Brain Repair and Integrative Neuroscience Program, The Research Institute of the McGill University Health Centre, Montréal, Québec, Canada. ^[1] Department of Neurology and Neurosurgery, Centre for Research in Neuroscience, McGill University, Québec, Canada; Brain Repair and Integrative Neuroscience Program, The Research Institute of the McGill University Health Centre, Montréal, Québec, Canada. Electronic address: wei-hsiang.huang@mcgill.ca. ^[2]

关键词 CRISPR activation Smith-Magenis syndrome gene therapy mouse models neurodevelopmental disorders retinoic acid induced 1

主题词小鼠(Mice);动物(Animals);Smith-Magenis综合征(Smith-Magenis Syndrome);反式激活因子类(Trans-Activators);依赖病毒(Dependovirus);单倍剂量不足(Haploinsufficiency);肥胖症(Obesity)

DOI 10.1016/j.jbc.2022.102728

PMID 36410433

发布时间 2023-02-02

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