Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial-Reference-Cited by-全球学者库

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

Published:2017-08 Issue:10097 Volume:390 Page:849-860
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Russell Stephen,Bennett Jean,Wellman Jennifer A,Chung Daniel C,Yu Zi-Fan,Tillman Amy,Wittes Janet,Pappas Julie,Elci Okan,McCague Sarah,Cross Dominique,Marshall Kathleen A,Walshire Jean,Kehoe Taylor L,Reichert Hannah,Davis Maria,Raffini Leslie,George Lindsey A,Hudson F Parker,Dingfield Laura,Zhu Xiaosong,Haller Julia A,Sohn Elliott H,Mahajan Vinit B,Pfeifer Wanda,Weckmann Michelle,Johnson Chris,Gewaily Dina,Drack Arlene,Stone Edwin,Wachtel Katie,Simonelli Francesca,Leroy Bart P,Wright J Fraser,High Katherine A,Maguire Albert M

Publisher

Elsevier BV

Subject

General Medicine

Reference42 articles.

1. Omics in ophthalmology: advances in genomics and precision medicine for Leber congenital amaurosis and age-related macular degeneration;den Hollander;Invest Ophthalmol Vis Sci,2016

2. Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark;Astuti;Eur J Hum Genet,2016

3. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis;Morimura;Proc Natl Acad Sci USA,1998

4. Genetics and phenotypes of RPE65 mutations in inherited retinal degeneration;Thompson;Invest Ophthalmol Vis Sci,2000

5. Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle;Redmond;Nat Genet,1998

Cited by 1179 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency;Molecular Therapy - Methods & Clinical Development;2024-03

2. Development and Validation of a Novel Mobility Test for Rod-Cone Dystrophies: From Reality to Virtual Reality;American Journal of Ophthalmology;2024-02

3. Innovative Genoceuticals in Human Gene Therapy Solutions: Challenges and Safe Clinical Trials of Orphan Gene Therapy Products;Current Gene Therapy;2024-02

4. Single Center Experience with Voretigene Neparvovec Gene Augmentation Therapy in RPE65 Mutation–Associated Inherited Retinal Degeneration in a Clinical Setting;Ophthalmology;2024-02

5. Novel objective outcomes to evaluate Voretigene Neparvovec treatment effects in clinical practice;Ophthalmology Retina;2024-02