Suppressing gain-of-function proteins via CRISPR/Cas9 system in SCA1 cells

SCAs are autosomal dominant neurodegenerative disorders caused by a gain-of-function protein with toxic activities, containing an expanded polyQ tract in the coding region. There are no treatments available to delay the onset, stop or slow down the progression of these pathologies. In this work we f...

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Detalles Bibliográficos
Autores principales: Pappadà, Mariangela, Bonuccelli, Ottavia, Buratto, Mattia, Fontana, Riccardo, Sicurella, Mariaconcetta, Caproni, Anna, Fuselli, Silvia, Benazzo, Andrea, Bertorelli, Roberto, De Sanctis, Veronica, Cavallerio, Paolo, Simioni, Valentina, Tugnoli, Valeria, Salvatori, Francesca, Marconi, Peggy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9700751/
https://www.ncbi.nlm.nih.gov/pubmed/36434031
http://dx.doi.org/10.1038/s41598-022-24299-y

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