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

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.

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Autores principales:	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
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2020
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/b91a2f099cbf40dd993de29571b96be7
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spelling	oai:doaj.org-article:b91a2f099cbf40dd993de29571b96be72021-12-02T16:45:30ZFrataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons10.1038/s41467-020-17954-32041-1723https://doaj.org/article/b91a2f099cbf40dd993de29571b96be72020-08-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-17954-3https://doaj.org/toc/2041-1723Friedreich’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.Pietro Giuseppe MazzaraSharon MuggeoMirko LuoniLuca MassiminoMattia ZaghiParisa Tajalli-Tehrani ValverdeSimone BruscoMatteo Jacopo MarziCecilia PalmaGaia ColasanteAngelo IannielliMarianna PaulisChiara CordiglieriSerena Gea GiannelliPaola PodiniCinzia GelleraFranco TaroniFrancesco NicassioMarco RasponiVania BroccoliNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-18 (2020)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q 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 Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
description	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.
format	article
author	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
author_facet	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
author_sort	Pietro Giuseppe Mazzara
title	Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
title_short	Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
title_full	Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
title_fullStr	Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
title_full_unstemmed	Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
title_sort	frataxin gene editing rescues friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons
publisher	Nature Portfolio
publishDate	2020
url	https://doaj.org/article/b91a2f099cbf40dd993de29571b96be7
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Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons

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