Molecular Therapies for Inherited Retinal Diseases—Current Standing, Opportunities and Challenges

由 Irene Vázquez-Domínguez, Alejandro Garanto, Rob W. J. Collin
出版 2019-08-01

Antisense Oligonucleotide Screening to Optimize the Rescue of the Splicing Defect Caused by the Recurrent Deep-Intronic <i>ABCA4</i> Variant c.4539+2001G&gt;A in Stargardt Disease

由 Alejandro Garanto, Lonneke Duijkers, Tomasz Z. Tomkiewicz, Rob W. J. Collin
出版 2019-06-01

Unexpected CEP290 mRNA splicing in a humanized knock-in mouse model for Leber congenital amaurosis.

由 Alejandro Garanto, Sylvia E C van Beersum, Theo A Peters, Ronald Roepman, Frans P M Cremers, Rob W J Collin
出版 2013-01-01

In or Out? New Insights on Exon Recognition through Splice-Site Interdependency

由 Mubeen Khan, Stéphanie S. Cornelis, Riccardo Sangermano, Iris J.M. Post, Amber Janssen Groesbeek, Jan Amsu, Christian Gilissen, Alejandro Garanto, Rob W.J. Collin, Frans P.M. Cremers
出版 2020-03-01