AAV Gene Therapy for Autosomal Dominant Optic Atrophy Caused by Mutation in the Opa1 Gene

被引：0
|
作者：
Stupay, Rachel M. [1 ]
Cascone, Alyna [1 ]
Padegimas, Linas [2 ]
Wille, Paul T. [1 ]
Kevany, Brian M. [3 ]
机构：
[1] Abeona Therapeut, Product Dev, Cleveland, OH USA
[2] Forge Biol, Grove City, OH USA
[3] Abeona Therapeut, Cleveland, OH USA
来源：
MOLECULAR THERAPY | 2023年 / 31卷 / 04期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
1478
引用
收藏
页码：696 / 697
页数：2
相关论文
共 50 条
  • [21] Identification of copy number variation in the gene for autosomal dominant optic atrophy, OPA1, in a Chinese pedigree
    Jin, X.
    Chen, Y. H.
    Liu, Z.
    Deng, Y.
    Li, N. N.
    Huang, H.
    Qi, M.
    Yi, X.
    Zhu, J.
    GENETICS AND MOLECULAR RESEARCH, 2015, 14 (03) : 10961 - 10972
  • [22] Optic disc morphology of patients with OPA1 autosomal dominant optic atrophy
    Votruba, M
    Thiselton, D
    Bhattacharya, SS
    BRITISH JOURNAL OF OPHTHALMOLOGY, 2003, 87 (01) : 48 - 53
  • [23] Development of an Optimized AAV Gene Therapy for Autosomal Dominant Optic Atrophy
    Liu, Ting
    Rong, Yu
    Cheng, Chao
    Zhang, Xin
    Luk, Alvin
    Li, Qiutang
    Li, Bin
    MOLECULAR THERAPY, 2022, 30 (04) : 251 - 251
  • [24] A recurrent deletion mutation in OPA1 causes autosomal dominant optic atrophy in a Chinese family
    Zhang, Liping
    Shi, Wei
    Song, Liming
    Zhang, Xiao
    Cheng, Lulu
    Wang, Yanfang
    Ge, Xianglian
    Li, Wei
    Zhang, Wei
    Min, Qingjie
    Jin, Zi-Bing
    Qu, Jia
    Gu, Feng
    SCIENTIFIC REPORTS, 2014, 4
  • [25] A splicing site OPA1 mutation associated with autosomal dominant optic atrophy in an Italian family
    Ranieri, M.
    Del Bo, R.
    Corti, S.
    Bordoni, A.
    Mancarella, G.
    Bresolin, N.
    Comi, G. P.
    JOURNAL OF NEUROLOGY, 2010, 257 : S205 - S205
  • [26] A recurrent deletion mutation in OPA1 causes autosomal dominant optic atrophy in a Chinese family
    Liping Zhang
    Wei Shi
    Liming Song
    Xiao Zhang
    Lulu Cheng
    Yanfang Wang
    Xianglian Ge
    Wei Li
    Wei Zhang
    Qingjie Min
    Zi-Bing Jin
    Jia Qu
    Feng Gu
    Scientific Reports, 4
  • [27] Mutation Screening of OPA1 in a cohort Chinese patients with Suspected Autosomal Dominant Optic Atrophy
    Xie, Yue
    Xiao, Ting
    Xu, Ke
    Zhang, Xiaohui
    Li, Yang
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2019, 60 (09)
  • [28] cDNA-based screening and analysis of the OPA1 gene in patients with autosomal dominant optic atrophy (adOA)
    Schimpf, S
    Schaich, S
    Wissinger, B
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2005, 46
  • [29] Dominant (Kjer's) optic atrophy associated with mutations in OPA1 gene
    Kelifova, S.
    Honzik, T.
    Tesarova, M.
    Kousal, B.
    Liskova, P.
    Havrankova, P.
    Kolarova, H.
    CESKA A SLOVENSKA NEUROLOGIE A NEUROCHIRURGIE, 2020, 83 (01) : 33 - 42
  • [30] Expression profile of OPA1, the gene associated with autosomal dominant optic atrophy in the developing and adult mammalian retina
    Aijaz, S
    Bhattacharya, SS
    Votruba, M
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2003, 44 : U375 - U375
12345