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Advanced genetic therapies for the treatment of Rett syndrome: state of the art and future perspectives

Loss and gain of functions mutations in the X-linked MECP2 (methyl-CpG-binding protein 2) gene are responsible for a set of generally severe neurological disorders that can affect both genders. In particular, Mecp2 deficiency is mainly associated with Rett syndrome (RTT) in girls, while duplication...

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Detalles Bibliográficos
Autores principales: Palmieri, Michela, Pozzer, Diego, Landsberger, Nicoletta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10248472/
https://www.ncbi.nlm.nih.gov/pubmed/37304036
http://dx.doi.org/10.3389/fnins.2023.1172805

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