Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C

por 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
Publicado 2023