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-同舟云学术

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 1313 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. Progressive nyctalopia and tunnel vision in a young man;Clinical Cases in Medical Retina;2025

2. Congenital blindness and retinopathy in a young girl;Clinical Cases in Medical Retina;2025

3. A Novel Method for Separating Full and Empty Adeno-Associated Viral Capsids Using Ultrafiltration;Membranes;2024-09-12

4. Clinical and Ocular Inflammatory Inhibitors of Viral-Based Gene Therapy of the Retina;Acta Microbiologica Hellenica;2024-09-11

5. Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial;Nature Communications;2024-09-10