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rAAV-CRISPRa therapy corrects Rai1 haploinsufficiency and rescues selective disease features in Smith-Magenis syndrome mice.
Chang, Hao-Cheng; Lee, Yu-Ju; Javed, Sehrish; Haque, Minza; Chang, Ya-Ting; Lin, Yu Cheng; Oram, Cameron; Huang, Wei-Hsiang.
Affiliation
  • Chang HC; 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.
  • Lee YJ; 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.
  • Javed S; 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.
  • Haque M; 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.
  • Chang YT; 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.
  • Lin YC; 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.
  • Oram C; 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.
  • Huang WH; 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
J Biol Chem ; 299(1): 102728, 2023 01.
Article in En | MEDLINE | ID: mdl-36410433
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Smith-Magenis Syndrome Limits: Animals Language: En Journal: J Biol Chem Year: 2023 Document type: Article Affiliation country: Canada Country of publication: United States
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Smith-Magenis Syndrome Limits: Animals Language: En Journal: J Biol Chem Year: 2023 Document type: Article Affiliation country: Canada Country of publication: United States