Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques

被引：6
|
作者：
Cho, Galaxy Y. [1 ,2 ,3 ,4 ]
Bolo, Kyle [5 ]
Park, Karen Sophia [1 ,2 ,3 ]
Sengillo, Jesse D. [6 ]
Tsang, Stephen H. [1 ,2 ,3 ,5 ,7 ,8 ]
机构：
[1] Jonas Childrens Vis Care, New York, NY 10032 USA
[2] Bernard & Shirlee Brown Glaucoma Lab, New York, NY 10032 USA
[3] Columbia Univ, Dept Ophthalmol, New York, NY 10027 USA
[4] Quinnipiac Univ, Frank H Netter MD Sch Med, North Haven, CT USA
[5] Columbia Univ, Vagelos Coll Phys & Surg, New York, NY 10032 USA
[6] Reading Hosp, Dept Internal Med, W Reading, PA USA
[7] Inst Human Nutr, Dept Pathol & Cell Biol, Stem Cell Initiat CSCI, New York, NY 10032 USA
[8] Columbia Univ, Med Ctr, Harkness Eye Inst, 635 West 165th St,Box 212, New York, NY 10032 USA
来源：
MOLECULAR DIAGNOSIS & THERAPY | 2019年 / 23卷 / 01期
基金：
美国国家卫生研究院;
关键词：
LEBER CONGENITAL AMAUROSIS; HEREDITARY OPTIC NEUROPATHY; ADENOASSOCIATED VIRUS; MACULAR DEGENERATION; SUBRETINAL INJECTION; RETINITIS-PIGMENTOSA; RPE65; MUTATIONS; IMMUNE-RESPONSE; OPEN-LABEL; FOLLOW-UP;
D O I：
10.1007/s40291-018-0377-1
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
Inherited retinal dystrophies cause progressive vision loss and are major contributors to blindness worldwide. Advances in gene therapy have brought molecular approaches into the realm of clinical trials for these incurable illnesses. Select phase I, II and III trials are complete and provide some promise in terms of functional outcomes and safety, although questions do remain over the durability of their effects and the prevalence of inflammatory reactions. This article reviews gene therapy as it can be applied to inherited retinal dystrophies, provides an update of results from recent clinical trials, and discusses the future prospects of gene therapy and genome surgery.
引用
收藏
页码：113 / 120
页数：8
相关论文
共 22 条
  • [1] Gene Therapy for Inherited Retinal Degeneration
    Arbabi, Amirmohsen
    Liu, Amelia
    Ameri, Hossein
    JOURNAL OF OCULAR PHARMACOLOGY AND THERAPEUTICS, 2019, 35 (02) : 79 - 97
  • [2] Gene-Based Therapeutics for Inherited Retinal Diseases
    Fenner, Beau J.
    Tan, Tien-En
    Barathi, Amutha Veluchamy
    Tun, Sai Bo Bo
    Yeo, Sia Wey
    Tsai, Andrew S. H.
    Lee, Shu Yen
    Cheung, Chui Ming Gemmy
    Chan, Choi Mun
    Mehta, Jodhbir S.
    Teo, Kelvin Y. C.
    FRONTIERS IN GENETICS, 2022, 12
  • [3] Gene-Based Therapeutics for Acquired Retinal Disease: Opportunities and Progress
    Tan, Tien-En
    Fenner, Beau James
    Barathi, Veluchamy Amutha
    Tun, Sai Bo Bo
    Wey, Yeo Sia
    Tsai, Andrew Shih Hsiang
    Su, Xinyi
    Lee, Shu Yen
    Cheung, Chui Ming Gemmy
    Wong, Tien Yin
    Mehta, Jodhbir Singh
    Teo, Kelvin Yi Chong
    FRONTIERS IN GENETICS, 2021, 12
  • [4] Gene and cell-based therapies for inherited retinal disorders: An update
    Sengillo, Jesse D.
    Justus, Sally
    Tsai, Yi-Ting
    Cabral, Thiago
    Tsang, Stephen H.
    AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS, 2016, 172 (04) : 349 - 366
  • [5] RPE based gene and cell therapy for inherited retinal diseases: A review
    Sanie-Jahromi, Fatemeh
    Nowroozzadeh, M. Hossein
    EXPERIMENTAL EYE RESEARCH, 2022, 217
  • [6] Focused Update on AAV-Based Gene Therapy Clinical Trials for Inherited Retinal Degeneration
    Fuller-Carter, Paula I.
    Basiri, Hamed
    Harvey, Alan R.
    Carvalho, Livia S.
    BIODRUGS, 2020, 34 (06) : 763 - 781
  • [7] Gene-based therapies for dominantly inherited retinopathies
    Farrar, G. J.
    Millington-Ward, S.
    Chadderton, N.
    Humphries, P.
    Kenna, P. F.
    GENE THERAPY, 2012, 19 (02) : 137 - 144
  • [8] 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
  • [9] Importance of Autoimmune Responses in Progression of Retinal Degeneration Initiated by Gene Mutations
    Adamus, Grazyna
    FRONTIERS IN MEDICINE, 2021, 8
  • [10] Non-invasive gene transfer by iontophoresis for therapy of an inherited retinal degeneration
    Souied, Eric H.
    Reid, Silvia N. M.
    Piri, Natik I.
    Lerner, Leonid E.
    Nusinowitz, Steven
    Farber, Debora B.
    EXPERIMENTAL EYE RESEARCH, 2008, 87 (03) : 168 - 175
123