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Safety and efficacy of genetic MECP2 supplementation in the R294X mouse model of Rett syndrome
Rett syndrome is a neurodevelopmental disorder caused predominantly by loss‐of‐function mutations in MECP2, encoding transcriptional modulator methyl‐CpG‐binding protein 2 (MeCP2). Although no disease‐modifying therapies exist at this time, some proposed therapeutic strategies aim to supplement the...
Autores principales: | Collins, Bridget E., Merritt, Jonathan K., Erickson, Kirsty R., Neul, Jeffrey L. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Blackwell Publishing Ltd
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8563491/ https://www.ncbi.nlm.nih.gov/pubmed/33942492 http://dx.doi.org/10.1111/gbb.12739 |
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