Article
Frataxin gene editing rescues Friedreich's ataxia pathology in dorsal root ganglia organoid-derived sensory neurons.
Nature communications - 21 Aug 2020
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
Abstract excerpt
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 differentiation of human iPSCs. Bulk and single-cell RNA sequencing show that DRG organoids present a transcriptional...
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