# Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

**Type:** Papers  
**Canonical URL:** https://scholariq.org/papers/efficacy-and-safety-of-voretigene-neparvovec-aav2-hrpe65v2-in-patients-with/

## Facts

| Field | Value |
| --- | --- |
| Author Names | Stephen R. Russell,Jean Bennett,Jennifer Wellman,Daniel C. Chung,Zi-Fan Yu,Amy Tillman,Janet Wittes,Julie Pappas,Okan U. Elci,Sarah McCague,Dominique Cross,Kathleen Marshall,Jean Walshire,Taylor Kehoe,Hannah Reichert,Maria Davis,Leslie Raffini,Lindsey A. George,Fleur Hudson,Laura Dingfield,Xiaosong Zhu,Julia A. Haller,Elliott H. Sohn,Vinit B. Mahajan,Wanda Pfeifer,Michelle T. Weckmann,Chris A. Johnson,Dina Y. Gewaily,Arlene V. Drack,Edwin M. Stone,Katie Wachtel,Francesca Simonelli,Bart P. Leroy,J. Fraser Wright,Katherine A. High,Albert M. Maguire |
| Citations | 1,923 |
| DOI | 10.1016/s0140-6736(17)31868-8 |
| Fields | Biochemistry, Genetics and Molecular Biology,Medicine,Neuroscience |
| Open Access | false |
| OA Status | closed |
| OpenAlex ID | https://openalex.org/W2736065260 |
| PMID | 28712537 |
| Type | article |
| Year | 2017 |

## Paper authors

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

## Paper journal

- [The Lancet](https://scholariq.org/journals/the-lancet/)

## 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/)
- [Photoreceptor and optogenetics research](https://scholariq.org/topics/photoreceptor-and-optogenetics-research/)
- [Retinal and Macular Surgery](https://scholariq.org/topics/retinal-and-macular-surgery/)

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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.