Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons

Friedreich’s ataxia (FRDA) is an autosomal-recessive neurodegenerative and cardiac disorder which occurs when transcription of the FXN gene is silenced due to an excessive expansion of GAA repeats into its first intron. Herein, we generate dorsal root ganglia organoids (DRG organoids) by in vitro di...

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Detalles Bibliográficos
Autores principales: Mazzara, Pietro Giuseppe, Muggeo, Sharon, Luoni, Mirko, Massimino, Luca, Zaghi, Mattia, Valverde, Parisa Tajalli-Tehrani, Brusco, Simone, Marzi, Matteo Jacopo, Palma, Cecilia, Colasante, Gaia, Iannielli, Angelo, Paulis, Marianna, Cordiglieri, Chiara, Giannelli, Serena Gea, Podini, Paola, Gellera, Cinzia, Taroni, Franco, Nicassio, Francesco, Rasponi, Marco, Broccoli, Vania
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7442818/
https://www.ncbi.nlm.nih.gov/pubmed/32826895
http://dx.doi.org/10.1038/s41467-020-17954-3

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