Frataxin gene editing rescues Friedreich's ataxia pathology in dorsal root ganglia organoid-derived sensory neurons.
Mazzara PG, Muggeo S, Luoni M, Massimino L, Zaghi M, Valverde PT, Brusco S, Marzi MJ, Palma C, Colasante G, Iannielli A, Paulis M, Cordiglieri C, Giannelli SG, Podini P, Gellera C, Taroni F, Nicassio F, Rasponi M, Broccoli V.
Mazzara PG, et al. Among authors: iannielli a.
Nat Commun. 2020 Aug 21;11(1):4178. doi: 10.1038/s41467-020-17954-3.
Nat Commun. 2020.
PMID: 32826895
Free PMC article.