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...

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Detalles Bibliográficos
Autores principales: Collins, Bridget E., Merritt, Jonathan K., Erickson, Kirsty R., Neul, Jeffrey L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Blackwell Publishing Ltd 2021
Materias:
Original Articles
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

Internet

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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