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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...
Autores principales: | Palmieri, Michela, Pozzer, Diego, Landsberger, Nicoletta |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2023
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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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