Article
Efficient clofilium tosylate-mediated rescue of POLG-related disease phenotypes in zebrafish.
Cell death & disease - 19 Jan 2021
Facchinello Nicola, Laquatra Claudio, Locatello Lisa, Beffagna Giorgia, Brañas Casas Raquel, Fornetto Chiara, Dinarello Alberto, Martorano Laura, Vettori Andrea, Risato Giovanni, Celeghin Rudy, Meneghetti Giacomo, Santoro Massimo Mattia, Delahodde Agnes, Vanzi Francesco, Rasola Andrea, Dalla Valle Luisa, Rasotto Maria Berica, Lodi Tiziana, Baruffini Enrico, Argenton Francesco, Tiso Natascia
Abstract excerpt
The DNA polymerase gamma (Polg) is a nuclear-encoded enzyme involved in DNA replication in animal mitochondria. In humans, mutations in the POLG gene underlie a set of mitochondrial diseases characterized by mitochondrial DNA (mtDNA) depletion or deletion and multiorgan defects, named POLG disorders, for which an effective therapy is still needed. By applying antisense strategies, ENU- and CRISPR/Cas9-based...
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