Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C
Titel:
Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C
Auteur:
Kaltak, Melita de Bruijn, Petra Piccolo, Davide Lee, Sang-Eun Dulla, Kalyan Hoogenboezem, Thomas Beumer, Wouter Webster, Andrew R. Collin, Rob W.J. Cheetham, Michael E. Platenburg, Gerard Swildens, Jim