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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Publicat a:Eur J Hum Genet
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: Springer International Publishing 2020
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Article
Accés en línia: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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