CRISPR/Cas9-based silencing of the ATXN1 gene in Spinocerebellar ataxia type 1 (SCA1) fibroblasts
Francesca Salvatori, Mariangela Pappadà, Mariaconcetta Sicurella, Mattia Buratto, Valentina Simioni, Valeria Tugnoli, Peggy Marconi
This study validates CRISPR/Cas9 as a therapeutic strategy for SCA1 by downregulating ATXN1 expression in patient fibroblasts with minimal off-target effects.
bioRxiv · Jul 4, 2020 · PreprintNeuroscience