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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Publicado no:Nat Commun
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7442818/
https://ncbi.nlm.nih.gov/pubmed/32826895
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-17954-3
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