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Site-directed RNA repair of endogenous Mecp2 RNA in neurons
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 M...
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| 出版年: | Proc Natl Acad Sci U S A |
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| 主要な著者: | , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
National Academy of Sciences
2017
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5676935/ https://ncbi.nlm.nih.gov/pubmed/29078406 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1715320114 |
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