Generation of Helper Plasmids Encoding Mutant Adeno-associated Virus Type 2 Capsid Proteins with Increased Resistance against Proteasomal Degradation by Naghmeh Ahmadiankia, Vajiheh Neshati, Zeinab Neshati, Jim Swildens, Antoine AF de Vries

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QR-1011 restores defective ABCA4 splicing caused by multiple severe ABCA4 variants underlying Stargardt disease by Melita Kaltak, Petra de Bruijn, Willemijn van Leeuwen, Gerard Platenburg, Frans P. M. Cremers, Rob W. J. Collin, Jim Swildens

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Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 function by Melita Kaltak, Rocio Blanco-Garavito, Laurie L. Molday, Claire-Marie Dhaenens, Eric E. Souied, Gerard Platenburg, Jim Swildens, Robert S. Molday, Frans P. M. Cremers

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

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Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis. by Wouter Beumer, Jim Swildens, Teresinha Leal, Sabrina Noel, Herma Anthonijsz, Geert van der Horst, Hester Kuiperij-Boersma, Marko Potman, Charlotte van Putten, Patricia Biasutto, Gerard Platenburg, Hugo de Jonge, Noreen Henig, Tita Ritsema

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Human embryonic and fetal mesenchymal stem cells differentiate toward three different cardiac lineages in contrast to their adult counterparts. by Arti A Ramkisoensing, Daniël A Pijnappels, Saïd F A Askar, Robert Passier, Jim Swildens, Marie José Goumans, Cindy I Schutte, Antoine A F de Vries, Sicco Scherjon, Christine L Mummery, Martin J Schalij, Douwe E Atsma

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