AAV-mediated FOXG1 gene editing in human Rett primary cells

Variations in the Forkhead Box G1 (FOXG1) gene cause FOXG1 syndrome spectrum, including the congenital variant of Rett syndrome, characterized by early onset of regression, Rett-like and jerky movements, and cortical visual impairment. Due to the largely unknown pathophysiological mechanisms downstr...

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Detalles Bibliográficos
Autores principales: Croci, Susanna, Carriero, Miriam Lucia, Capitani, Katia, Daga, Sergio, Donati, Francesco, Papa, Filomena Tiziana, Frullanti, Elisa, Lopergolo, Diego, Lamacchia, Vittoria, Tita, Rossella, Giliberti, Annarita, Benetti, Elisa, Niccheri, Francesca, Furini, Simone, Lo Rizzo, Caterina, Conticello, Silvestro Giovanni, Renieri, Alessandra, Meloni, Ilaria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7608362/
https://www.ncbi.nlm.nih.gov/pubmed/32541681
http://dx.doi.org/10.1038/s41431-020-0652-6

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