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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Detaylı Bibliyografya
Yayımlandı:Proc Natl Acad Sci U S A
Asıl Yazarlar: Sinnamon, John R., Kim, Susan Y., Corson, Glen M., Song, Zhen, Nakai, Hiroyuki, Adelman, John P., Mandel, Gail
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: National Academy of Sciences 2017
Konular:
PNAS Plus
Online Erişim: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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