Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells

by Florent Porquet, Lin Weidong, Kévin Jehasse, Hélène Gazon, Maria Kondili, Silvia Blacher, Laurent Massotte, Emmannuel Di Valentin, Denis Furling, Nicolas Albert Gillet, Arnaud François Klein, Vincent Seutin, Luc Willems
Published 2023-06-01