Nature Communications (Aug 2020)
Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
- Pietro Giuseppe Mazzara,
- Sharon Muggeo,
- Mirko Luoni,
- Luca Massimino,
- Mattia Zaghi,
- Parisa Tajalli-Tehrani Valverde,
- Simone Brusco,
- Matteo Jacopo Marzi,
- Cecilia Palma,
- Gaia Colasante,
- Angelo Iannielli,
- Marianna Paulis,
- Chiara Cordiglieri,
- Serena Gea Giannelli,
- Paola Podini,
- Cinzia Gellera,
- Franco Taroni,
- Francesco Nicassio,
- Marco Rasponi,
- Vania Broccoli
Affiliations
- Pietro Giuseppe Mazzara
- Division of Neuroscience, San Raffaele Scientific Institute
- Sharon Muggeo
- Division of Neuroscience, San Raffaele Scientific Institute
- Mirko Luoni
- Division of Neuroscience, San Raffaele Scientific Institute
- Luca Massimino
- Division of Neuroscience, San Raffaele Scientific Institute
- Mattia Zaghi
- Division of Neuroscience, San Raffaele Scientific Institute
- Parisa Tajalli-Tehrani Valverde
- Division of Neuroscience, San Raffaele Scientific Institute
- Simone Brusco
- Division of Neuroscience, San Raffaele Scientific Institute
- Matteo Jacopo Marzi
- Center for Genomic Science of IIT@SEMM, Istituto Italiano di Tecnologia (IIT)
- Cecilia Palma
- Department of Electronics, Information & Bioengineering, Politecnico di Milano
- Gaia Colasante
- Division of Neuroscience, San Raffaele Scientific Institute
- Angelo Iannielli
- Division of Neuroscience, San Raffaele Scientific Institute
- Marianna Paulis
- Humanitas Clinical and Research Center
- Chiara Cordiglieri
- National Institute of Molecular Genetics “Romeo e Enrica Invernizzi” - INGM
- Serena Gea Giannelli
- Division of Neuroscience, San Raffaele Scientific Institute
- Paola Podini
- Division of Neuroscience, San Raffaele Scientific Institute
- Cinzia Gellera
- Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico Carlo Besta
- Franco Taroni
- Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico Carlo Besta
- Francesco Nicassio
- Center for Genomic Science of IIT@SEMM, Istituto Italiano di Tecnologia (IIT)
- Marco Rasponi
- Department of Electronics, Information & Bioengineering, Politecnico di Milano
- Vania Broccoli
- Division of Neuroscience, San Raffaele Scientific Institute
- DOI
- https://doi.org/10.1038/s41467-020-17954-3
- Journal volume & issue
-
Vol. 11,
no. 1
pp. 1 – 18
Abstract
Friedreich’s ataxia (FRDA) is an autosomal-recessive disorder. Here the authors describe a DRG organoid from patient derived-neurons and co-culture with muscle cells to mimic the disorder in vitro and demonstrate potential correction of the phenotype by CRISPR based editing.
