Caroline Van Cauwenbergh

Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect

Artur V. Cideciyan, Samuel G. Jacobson, Arlene V. Drack et al. · Nature Medicine · 2018 · 216 citations · Full text

Medicine, Ophthalmology, Vision Science +15

ABCA4-associated disease as a model for missing heritability in autosomal recessive disorders: novel noncoding splice, cis-regulatory, structural, and recurrent hypomorphic variants

Alejandro Garanto, Riccardo Sangermano, Sarah Naessens et al. · Genetics in Medicine · 2019 · 163 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

Stephen R. Russell, Arlene V. Drack, Artur V. Cideciyan et al. · Nature Medicine · 2022 · 130 citations · Full text

Medicine, Ophthalmology, Pharmacotherapy +13

Charting Extracellular Transcriptomes in The Human Biofluid RNA Atlas

Eva Hulstaert, Annelien Morlion, Francisco Avila Cobos et al. · Cell Reports · 2020 · 94 citations · Full text

Life Sciences, Biomedical Sciences, Molecular Biology +14

X-Linked Retinoschisis

Leo C. Hahn, Mary J. van Schooneveld, Nieneke L. Wesseling et al. · Ophthalmology · 2021 · 77 citations · Full text

Medicine, Ophthalmology, Vision Science +17