Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
Titel:
Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
Auteur:
Dulla, Kalyan Aguila, Monica Lane, Amelia Jovanovic, Katarina Parfitt, David A. Schulkens, Iris Chan, Hee Lam Schmidt, Iris Beumer, Wouter Vorthoren, Lars Collin, Rob W.J. Garanto, Alejandro Duijkers, Lonneke Brugulat-Panes, Anna Semo, Ma’ayan Vugler, Anthony A. Biasutto, Patricia Adamson, Peter Cheetham, Michael E.