Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65 (vol 23, pg 857, 2016)

被引：0
|
作者：
Georgiadis, A.
Duran, Y.
Ribeiro, J.
Abelleira-Hervas, L.
Robbie, S. J.
Sunkel-Laing, B.
Fourali, S.
Gonzalez-Cordero, A.
Cristante, E.
Michaelides, M.
Bainbridge, J. W. B.
Smith, A. J.
Ali, R. R.
机构：
[1] UCL Institute of Ophthalmology,Department of Genetics
[2] NIHR Biomedical Research Centre at Moorfields Eye Hospital,undefined
来源：
GENE THERAPY | 2024年 / 31卷 / 9-10期
关键词：
D O I：
10.1038/s41434-024-00463-z
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
引用
收藏
页码：524 / 524
页数：1
相关论文
共 38 条
  • [21] Gene therapy for vision restoration in patients with Leber congenital amaurosis (LCA) due to RPE65 gene mutations: beginning the phase IV trial
    Francisco Chacon-Camacho, Oscar
    Carlos Zenteno, Juan
    GACETA MEDICA DE MEXICO, 2017, 153 (02): : 276 - 278
  • [22] Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
    Maguire, Albert M.
    High, Katherine A.
    Auricchio, Alberta
    Wright, J. Fraser
    Pierce, Eric A.
    Testa, Francesco
    Mingozzi, Federico
    Bennicelli, Jeannette L.
    Ying, Gui-shuang
    Rossi, Settimio
    Fulton, Ann
    Marshall, Kathleen A.
    Banfi, Sandra
    Chung, Daniel C.
    Morgan, Jessica I. W.
    Hauck, Bernd
    Zelenaia, Olga
    Zhu, Xiaosong
    Raffini, Leslie
    Coppieters, Frauke
    De Baere, Elfride
    Shindler, Kenneth S.
    Volpe, Nicholas J.
    Surace, Enrico M.
    Acerra, Carmela
    Lyubarsky, Arkady
    Redmond, T. Michael
    Stone, Edwin
    Sun, Junwei
    McDonnell, Jennifer Wellman
    Leroy, Bart P.
    Simonelli, Francesca
    Bennett, Jean
    LANCET, 2009, 374 (9701): : 1597 - 1605
  • [23] Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis
    Pang, JJ
    Chang, B
    Kumar, A
    Nusinowitz, S
    Noorwez, SM
    Li, J
    Rani, A
    Foster, TC
    Chiodo, VA
    Doyle, T
    Li, HS
    Malhotra, R
    Teusner, JT
    McDowell, JH
    Min, SH
    Li, QH
    Kaushal, S
    Hauswirth, WW
    MOLECULAR THERAPY, 2006, 13 (03) : 565 - 572
  • [24] Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years
    Jacobson, Samuel G.
    Cideciyan, Artur V.
    Ratnakaram, Ramakrishna
    Heon, Elise
    Schwartz, Sharon B.
    Roman, Alejandro J.
    Peden, Marc C.
    Aleman, Tomas S.
    Boye, Sanford L.
    Sumaroka, Alexander
    Conlon, Thomas J.
    Calcedo, Roberto
    Pang, Ji-Jing
    Erger, Kirsten E.
    Olivares, Melani B.
    Mullins, Cristina L.
    Swider, Malgorzata
    Kaushal, Shalesh
    Feuer, William J.
    Iannaccone, Alessandro
    Fishman, Gerald A.
    Stone, Edwin M.
    Byrne, Barry J.
    Hauswirth, William W.
    ARCHIVES OF OPHTHALMOLOGY, 2012, 130 (01) : 9 - 24
  • [25] Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. (vol 374, pg 1597, 2009)
    Maguire, A. M.
    High, K. A.
    Auricchio, A.
    LANCET, 2010, 375 (9708): : 30 - 30
  • [26] Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors (vol 18, pg 2099, 2009)
    不详
    HUMAN MOLECULAR GENETICS, 2023, 32 (24) : 3391 - 3393
  • [27] Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors (vol 18, pg 2099, 2009)
    Tan, Mei Hong
    Smith, Alexander J.
    Pawlyk, Basil
    Xu, Xiaoyun
    Liu, Xiaoqing
    Bainbridge, James B.
    Basche, Mark
    McIntosh, Jenny
    Tran, Hoai Viet
    Nathwani, Amit
    Li, Tiansen
    Ali, Robin R.
    HUMAN MOLECULAR GENETICS, 2010, 19 (04) : 735 - 735
  • [28] Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors (vol 18, pg 2099, 2009)
    Tan, Mei Hong
    Smith, Alexander J.
    Pawlyk, Basil
    Xu, Xiaoyun
    Liu, Xiaoqing
    Bainbridge, James B.
    Basche, Mark
    McIntosh, Jenny
    Tran, Hoai Viet
    Nathwani, Amit
    Li, Tiansen
    Ali, Robin R.
    HUMAN MOLECULAR GENETICS, 2024, 33 (10) : 931 - 933
  • [29] Gene Therapy Rescues Cone Structure and Function in the 3-Month-Old rd12 Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis
    Li, Xia
    Li, Wensheng
    Dai, Xufeng
    Kong, Fansheng
    Zheng, Qinxiang
    Zhou, Xiangtian
    Lue, Fan
    Chang, Bo
    Rohrer, Baerbel
    Hauswirth, William. W.
    Qu, Jia
    Pang, Ji-jing
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2011, 52 (01) : 7 - 15
  • [30] Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors
    Tan, Mei Hong
    Smith, Alexander J.
    Pawlyk, Basil
    Xu, Xiaoyun
    Liu, Xiaoqing
    Bainbridge, James B.
    Basche, Mark
    McIntosh, Jenny
    Tran, Hoai Viet
    Nathwani, Amit
    Li, Tiansen
    Ali, Robin R.
    HUMAN MOLECULAR GENETICS, 2009, 18 (12) : 2099 - 2114
1234