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High rate of HDR in gene editing of p.(Thr158Met) MECP2 mutational hotspot
Rett syndrome is a progressive neurodevelopmental disorder which affects almost exclusively girls, caused by variants in MECP2 gene. Effective therapies for this devastating disorder are not yet available and the need for tight regulation of MECP2 expression for brain to properly function makes gene...
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| Udgivet i: | Eur J Hum Genet |
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| Main Authors: | , , , , , , , , , , , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Springer International Publishing
2020
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7609331/ https://ncbi.nlm.nih.gov/pubmed/32332872 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41431-020-0624-x |
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