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

**Type:** Papers  
**Canonical URL:** https://scholariq.org/papers/intravitreal-antisense-oligonucleotide-sepofarsen-in-leber-congenital-amaurosis/

## Facts

| Field | Value |
| --- | --- |
| Author Names | Stephen R. Russell,Arlene V. Drack,Artur V. Cideciyan,Samuel G. Jacobson,Bart P. Leroy,Caroline Van Cauwenbergh,Allen C. Ho,Alina V. Dumitrescu,Ian C. Han,M. Carmen Marcos Martin,Wanda Pfeifer,Elliott H. Sohn,Jean Walshire,Alexandra V. Garafalo,Arun K. Krishnan,Christian A. Powers,Alexander Sumaroka,Alejandro J. Román,Eva Vanhonsebrouck,Eltanara A. Jones,Fanny Nerinckx,Julie De Zaeytijd,Rob W.J. Collin,Carel B. Hoyng,Peter Adamson,Michael E. Cheetham,Michael R. Schwartz,Wilhelmina den Hollander,Friedrich Asmus,Gerard Platenburg,David M. Rodman,Aniz Girach |
| Citations | 134 |
| DOI | 10.1038/s41591-022-01755-w |
| Fields | Biochemistry, Genetics and Molecular Biology,Medicine |
| Open Access | true |
| OA Status | hybrid |
| OA URL | https://www.nature.com/articles/s41591-022-01755-w.pdf |
| OpenAlex ID | https://openalex.org/W4225983615 |
| PMID | 35379979 |
| Type | article |
| Year | 2022 |

## Paper authors

- [Wanda Pfeifer](https://scholariq.org/researchers/wanda-pfeifer/)

## Paper journal

- [Nature Medicine](https://scholariq.org/journals/nature-medicine/)

## Paper primary topic

- [Retinal Development and Disorders](https://scholariq.org/topics/retinal-development-and-disorders/)

## Paper topics

- [Retinal Development and Disorders](https://scholariq.org/topics/retinal-development-and-disorders/)
- [Retinal Diseases and Treatments](https://scholariq.org/topics/retinal-diseases-and-treatments/)
- [Ocular Disorders and Treatments](https://scholariq.org/topics/ocular-disorders-and-treatments/)

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Source: ScholarIQ — public research metadata, principally OpenAlex. See https://scholariq.org/sources/ for provenance and https://scholariq.org/methodology/ for what these figures mean.