Alexandra V. Garafalo

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 · 209 citations · Full text

Leber Congenital Amaurosis, Photoreceptor Cilium Defect, Ocular Disease +10

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 · 121 citations · Full text

Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report

Artur V. Cideciyan, Samuel G. Jacobson, Allen C. Ho et al. · Nature Medicine · 2021 · 61 citations · Full text

Ocular Disease, Vitreous Body, Ophthalmology +5

EYS Mutations Causing Autosomal Recessive Retinitis Pigmentosa: Changes of Retinal Structure and Function with Disease Progression

David B. McGuigan, Elise Héon, Artur V. Cideciyan et al. · Genes · 2017 · 55 citations · Full text

Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations

Samuel G. Jacobson, Artur V. Cideciyan, Allen C. Ho et al. · iScience · 2021 · 43 citations · Full text