Conference Proceedings

A Dominant-Negative COL6A1 Pseudoexon Insertion Is Skippable Using Splice-Modulating Oligonucleotides

Veronique Bolduc, A Reghan Foley, Sandra Donkervoort, Ying Hu, Beryl B Cummings, Monkol Lek, Apurva Sarathy, Katherine Sizov, Herimela Solomon Degefa, Raimund Wagener, Grant W Hennig, Eric Hanssen, Shireen R Lamande, Francesco Muntoni, Steve D Wilton, Daniel G MacArthur, Carsten G Bonnemann

MOLECULAR THERAPY | CELL PRESS | Published : 2017