Article
Site-directed RNA repair of endogenous Mecp2 RNA in neurons.
Proceedings of the National Academy of Sciences of the United States of America - 31 Oct 2017
Sinnamon John R, Kim Susan Y, Corson Glen M, Song Zhen, Nakai Hiroyuki, Adelman John P, Mandel Gail
Abstract excerpt
Rett syndrome (RTT) is a debilitating neurological disorder caused by mutations in the gene encoding the transcription factor Methyl CpG Binding Protein 2 (MECP2). A distinct disorder results from MECP2 gene duplication, suggesting that therapeutic approaches must restore close to normal levels of MECP2. Here, we apply the approach of site-directed RNA editing to repair, at the mRNA level, a disease-causing...
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