Gene therapy in retinal diseases: A review

被引：30

作者：

Dhurandhar, Deven ^{[1
]}

Sahoo, Niroj Kumar ^{[2
]}

Mariappan, Indumathi ^{[3
]}

Narayanan, Raja ^{[2
]}

机构：

[1] LV Prasad Eye Inst, Retina & Uveitis Dept, GMR Varalakshmi Campus, Visakhapatnam, Andhra Pradesh, India

[2] LV Prasad Eye Inst, Smt Kanuri Santhamma Ctr VitreoRetinal Dis, Kallam Anji Reddy Campus, Hyderabad, Telangana, India

[3] LV Prasad Eye Inst, Ctr Ocular Regenerat, Hyderabad Eye Res Fdn, Kallam Anji Reddy Campus, Hyderabad, Telangana, India

来源：

INDIAN JOURNAL OF OPHTHALMOLOGY | 2021年 / 69卷 / 09期

关键词：

Adeno-associated viral vectors (AAVs); age-related macular degeneration (AMD); gene delivery systems; gene therapy; inherited retinal diseases; Luxturna; non-inherited retinal diseases; visual cycle; voretigene neparvovec; LEBER CONGENITAL AMAUROSIS; MACULAR DEGENERATION; BINDING PROTEIN; OPEN-LABEL; MUTATION; VECTOR; PHENOTYPE; DYSTROPHY; NEOVASCULARIZATION; PHASE-1;

D O I：

10.4103/ijo.IJO_3117_20

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

Over 2 million people worldwide are suffering from gene-related retinal diseases, inherited or acquired, and over 270 genes have been identified which are found to be responsible for these conditions. This review article touches upon the mechanisms of gene therapy, various enzymes of the visual cycle responsible for different genetic diseases, Luxturna-the first US Food and Drug Administration (FDA)-approved therapeutic gene product, and several ongoing trials of gene therapy for age-related macular degeneration. Gene therapy has tremendous potential for retinal conditions due to its ease of accessibility, immune-privileged status, and tight blood-retinal barriers, limiting systemic side effects of the drug. In recent years, advances in gene therapy in retinal conditions have increasing significantly, with progress in cell-specific targeting and transduction efficiency of gene products through the use of adeno-associated viral vectors (AAVs), suggesting that even greater success in future clinical trials is possible.

引用

页码：2257 / 2265

页数：9

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