| Delivery of oligonucleotide‐based therapeutics: challenges and opportunities SM Hammond, A Aartsma‐Rus, S Alves, SE Borgos, RAM Buijsen, ... EMBO molecular medicine 13 (4), e13243, 2021 | 207 | 2021 |
| In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery A Garanto, DC Chung, L Duijkers, JC Corral-Serrano, M Messchaert, ... Human molecular genetics 25 (12), 2552-2563, 2016 | 205 | 2016 |
| Deep-intronic ABCA4 variants explain missing heritability in Stargardt disease and allow correction of splice defects by antisense oligonucleotides R Sangermano, A Garanto, M Khan, EH Runhart, M Bauwens, NM Bax, ... Genetics in Medicine 21 (8), 1751-1760, 2019 | 169 | 2019 |
| ABCA4 midigenes reveal the full splice spectrum of all reported noncanonical splice site variants in Stargardt disease R Sangermano, M Khan, SS Cornelis, V Richelle, S Albert, A Garanto, ... Genome research 28 (1), 100-110, 2018 | 161 | 2018 |
| ABCA4-associated disease as a model for missing heritability in autosomal recessive disorders: novel noncoding splice, cis-regulatory, structural, and recurrent hypomorphic … M Bauwens, A Garanto, R Sangermano, S Naessens, N Weisschuh, ... Genetics in Medicine 21 (8), 1761-1771, 2019 | 141 | 2019 |
| Unexpected CEP290 mRNA splicing in a humanized knock-in mouse model for Leber congenital amaurosis A Garanto, SEC van Beersum, TA Peters, R Roepman, FPM Cremers, ... PLoS One 8 (11), e79369, 2013 | 135 | 2013 |
| Splice-modulating oligonucleotide QR-110 restores CEP290 mRNA and function in human c. 2991+ 1655A> G LCA10 models K Dulla, M Aguila, A Lane, K Jovanovic, DA Parfitt, I Schulkens, HL Chan, ... Molecular Therapy-Nucleic Acids 12, 730-740, 2018 | 134 | 2018 |
| Identification and rescue of splice defects caused by two neighboring deep-intronic ABCA4 mutations underlying Stargardt disease S Albert, A Garanto, R Sangermano, M Khan, NM Bax, CB Hoyng, ... The American Journal of Human Genetics 102 (4), 517-527, 2018 | 127 | 2018 |
| Photoreceptor progenitor mRNA analysis reveals exon skipping resulting from the ABCA4 c. 5461-10T→ C mutation in Stargardt disease R Sangermano, NM Bax, M Bauwens, LI Van den Born, E De Baere, ... Ophthalmology 123 (6), 1375-1385, 2016 | 121 | 2016 |
| Antisense oligonucleotide-based splice correction for USH2A-associated retinal degeneration caused by a frequent deep-intronic mutation RWN Slijkerman, C Vaché, M Dona, G García-García, M Claustres, ... Molecular Therapy-Nucleic Acids 5, 2016 | 115 | 2016 |
| Molecular therapies for inherited retinal diseases—current standing, opportunities and challenges I Vázquez-Domínguez, A Garanto, RWJ Collin Genes 10 (9), 654, 2019 | 72 | 2019 |
| Overexpression of CERKL, a gene responsible for retinitis pigmentosa in humans, protects cells from apoptosis induced by oxidative stress M Tuson, A Garanto, R Gonzàlez-Duarte, G Marfany Molecular vision 15, 168, 2009 | 72 | 2009 |
| Detailed phenotyping and therapeutic strategies for intronic ABCA4 variants in Stargardt disease M Khan, G Arno, A Fakin, DA Parfitt, PPA Dhooge, S Albert, NM Bax, ... Molecular Therapy-Nucleic Acids 21, 412-427, 2020 | 62 | 2020 |
| Antisense Oligonucleotide Screening to Optimize the Rescue of the Splicing Defect Caused by the Recurrent Deep-Intronic ABCA4 Variant c.4539+2001G>A in … A Garanto, L Duijkers, TZ Tomkiewicz, RWJ Collin Genes 10 (6), 452, 2019 | 60 | 2019 |
| Whole-exome sequencing reveals ZNF408 as a new gene associated with autosomal recessive retinitis pigmentosa with vitreal alterations A Avila-Fernandez, R Perez-Carro, M Corton, MI Lopez-Molina, ... Human molecular genetics 24 (14), 4037-4048, 2015 | 52 | 2015 |
| PCARE and WASF3 regulate ciliary F-actin assembly that is required for the initiation of photoreceptor outer segment disk formation JC Corral-Serrano, IJC Lamers, J van Reeuwijk, L Duijkers, ... Proceedings of the National Academy of Sciences 117 (18), 9922-9931, 2020 | 51 | 2020 |
| Implications of genetic variation in the complement system in age-related macular degeneration S de Jong, G Gagliardi, A Garanto, A de Breuk, YTE Lechanteur, S Katti, ... Progress in Retinal and Eye Research 84, 100952, 2021 | 47 | 2021 |
| Therapeutic effects of the mitochondrial ROS-redox modulator KH176 in a mammalian model of Leigh Disease R de Haas, D Das, A Garanto, HG Renkema, R Greupink, ... Scientific reports 7 (1), 11733, 2017 | 45 | 2017 |
| Specific sphingolipid content decrease in Cerkl knockdown mouse retinas A Garanto, NA Mandal, M Egido-Gabas, G Marfany, G Fabrias, ... Experimental eye research 110, 96-106, 2013 | 45 | 2013 |
| A look into retinal organoids: methods, analytical techniques, and applications TAV Afanasyeva, JC Corral-Serrano, A Garanto, R Roepman, ... Cellular and Molecular Life Sciences 78, 6505-6532, 2021 | 43 | 2021 |
