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Gene Editing to the Rescue:Reversal of Social Deficits Associated with MECP2 Duplication 被引量:1

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摘要 Methyl-CpG-binding protein 2 (MeCP2) is broadly recognized as the genetic cause of Rett Syndrome (RTT),a devastating neurodevelopmental disorder with the progressive loss in motor skills and speech that is found almost exclusively in young girls [1,2].Over 95%of RTT patients carry loss-of-function mutations in the X-linked MECP2gene,while the same mutations in males largely lead to infantile death.
出处 《Neuroscience Bulletin》 SCIE CAS CSCD 2020年第6期567-569,共3页 神经科学通报(英文版)
关键词 MECP2 MECP2 RESCUE
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