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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Publicado en:Eur J Hum Genet
Main Authors: 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: Artigo
Idioma:Inglês
Publicado: Springer International Publishing 2020
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7608362/
https://ncbi.nlm.nih.gov/pubmed/32541681
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41431-020-0652-6
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