# 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

*PRI Rank #12 · Topics and Trends in Most Cited Retinal Development and Disorders Papers, Class of 2026*

*Canonical URL: https://pri.pepkio.com/top-papers/retinal-development-and-disorders/2026/rank-12*

| Field | Value |
| --- | --- |
| Rank | #12 |
| 18m citations | 28 |
| Journal | The Lancet |
| Year | 2024 |
| DOI | 10.1016/s0140-6736(24)01447-8 |
| Corresponding authors | Paul Yang, Artur V. Cideciyan |
| Institution | Oregon Health & Science University, United States |

**Ranking page:** [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)

**Paper link:** [10.1016/s0140-6736(24)01447-8](https://doi.org/10.1016/s0140-6736(24)01447-8)

## 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

## Cite this ranking

```
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 2026-10-28.

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
```