Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study

🏆 Top 20📊 OpenAlex

Most Cited Paper

in Retinal Development and Disorders

18 Months Post-Publication

Class of 2026

“Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study”

AuthorsPaul Yang et al.

Paul Yang, Artur V. Cideciyan

JournalThe Lancet

Oregon Health & Science University

Citations28

DOI10.1016/s0140-6736(24)01447-8

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Topics

ATSN-101 · Leber congenital amaurosis · Retinal guanylate cyclase · biallelic mutations · Phase 1/2 clinical trial · dose escalation · unilateral administration · gene therapy · Retinal guanylate cyclase · Inherited retinal degeneration · Subretinal injection · visual function · retinal structure · adeno-associated viral vector

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Pepkio Research Index (PRI). Topics and Trends in Most Cited Retinal Development and Disorders Papers, Class of 2026. https://pri.pepkio.com/top-papers/retinal-development-and-disorders/2026. Accessed 2026-10-24.

Methodology
Zheng Su, Tinsley Li, Thematic Shifts in Early-High-Impact Cancer Genomics and Diagnostics Research: A Bibliometric and Semantic Analysis. bioRxiv 2026.07.04.736459; doi: https://doi.org/10.64898/2026.07.04.736459