Gene Therapy for Retinal Degeneration

被引：59
|
作者：
Apte, Rajendra S. [1 ]
机构：
[1] Washington Univ, Sch Med, 660 South Euclid Ave,Box 8096, St Louis, MO 63110 USA
来源：
CELL | 2018年 / 173卷 / 01期
关键词：
D O I：
10.1016/j.cell.2018.03.021
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：
Biallelic mutations in the RPE65 gene are associated with inherited retinal degenerations/dystrophies (IRD) and disrupt the visual cycle, leading to loss of vision. A new adenoviral vector-based gene therapy surgically delivered to retinal cells provides normal human RPE65 protein that can restore the visual cycle and some vision.
引用
收藏
页码：5 / 5
页数：1
相关论文
共 50 条
  • [31] Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy
    Robin R. Ali
    Gian-Marco Sarra
    Clare Stephens
    Mahesh de Alwis
    James W.B. Bainbridge
    Peter M. Munro
    Sascha Fauser
    Martin B. Reichel
    Christine Kinnon
    David M. Hunt
    Shomi S. Bhattacharya
    Adrian J. Thrasher
    Nature Genetics, 2000, 25 : 306 - 310
  • [32] Gene therapy to increase and maintain light sensitivity in cones during retinal degeneration
    Simon, C.
    Dalkara, D.
    HUMAN GENE THERAPY, 2019, 30 (11) : A111 - A111
  • [33] TAUFON THERAPY OF RETINAL PIGMENTARY DEGENERATION
    VODOVOZOV, AM
    GLOTOVA, NM
    YARTSEV, EI
    VARNOVITSKY, AM
    BORISKINA, LN
    VESTNIK OFTALMOLOGII, 1986, (06) : 38 - 39
  • [34] Stem cell therapy for retinal degeneration
    Ali, Robin
    HUMAN GENE THERAPY, 2013, 24 (05) : A11 - A11
  • [35] Anti-Oxidant Gene Delivery to Retinal Epithelial Cells: A Gene Therapy Approach for Macular Degeneration
    McGinley, Lisa
    Cosgrave, Gillian
    Duffy, Aoife
    O'Brien, Timothy
    Strappe, Padraig M.
    MOLECULAR THERAPY, 2006, 13 : S340 - S341
  • [36] Gene Therapy for Rdh12-Associated Retinal Diseases Helps to Delay Retinal Degeneration and Vision Loss
    Bian, Jiaxin
    Chen, Hongyu
    Sun, Junhui
    Cao, Yuqing
    An, Jianhong
    Pan, Qing
    Qi, Ming
    DRUG DESIGN DEVELOPMENT AND THERAPY, 2021, 15 : 3581 - 3591
  • [37] Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration
    Greenwald, Scott H.
    Brown, Emily E.
    Scandura, Michael J.
    Hennessey, Erin
    Farmer, Raymond
    Pawlyk, Basil S.
    Xiao, Ru
    Vandenberghe, Luk H.
    Pierce, Eric A.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 18 : 582 - 594
  • [38] Gene augmentation therapy for NMNAT1-associated retinal degeneration: Promise and challenges
    Greenwald, Scott H.
    Pawlyk, Basil S.
    Hennessey, Erin
    Scandura, Michael J.
    Xiao, Ru
    Vandenberghe, Luk H.
    Pierce, Eric A.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2019, 60 (09)
  • [39] Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A
    Lopes, Vanda S.
    Williams, David S.
    COLD SPRING HARBOR PERSPECTIVES IN MEDICINE, 2015, 5 (06): : 1 - 10
  • [40] Patient-reported outcome measures in inherited retinal degeneration gene therapy trials
    Lacy, Gabrielle D.
    Abalem, Maria Fernanda
    Musch, David C.
    Jayasundera, Kanishka T.
    OPHTHALMIC GENETICS, 2020, 41 (01) : 1 - 6
12345