Gene-Based Therapeutics for Inherited Retinal Diseases

被引:44
作者
Fenner, Beau J. [1 ,2 ,3 ]
Tan, Tien-En [1 ,2 ,3 ]
Barathi, Amutha Veluchamy [2 ]
Tun, Sai Bo Bo [2 ]
Yeo, Sia Wey [2 ]
Tsai, Andrew S. H. [1 ,2 ,3 ]
Lee, Shu Yen [1 ,2 ,3 ]
Cheung, Chui Ming Gemmy [1 ,2 ,3 ]
Chan, Choi Mun [1 ,2 ,3 ]
Mehta, Jodhbir S. [1 ,2 ,3 ,4 ,5 ]
Teo, Kelvin Y. C. [1 ,2 ,3 ]
机构
[1] Singapore Natl Eye Ctr, Singapore, Singapore
[2] Singapore Eye Res Inst, Singapore, Singapore
[3] Duke NUS Grad Med Sch, Ophthalmol & Visual Sci Acad Clin Programme, Singapore, Singapore
[4] Nanyang Technol Univ, Sch Mat Sci & Engn, Singapore, Singapore
[5] Natl Univ Singapore, Yong Loo Lin Sch Med, Dept Ophthalmol, Singapore, Singapore
关键词
inherited retinal diseases (IRDs); gene tharapy; adeno-associated virus; RNA editing; CRISPR; optogenetic; antisense oligonucleotides; retina; DOMINANT RETINITIS-PIGMENTOSA; ANTISENSE OLIGONUCLEOTIDE; VORETIGENE NEPARVOVEC; LARGE COHORT; AAV VECTORS; THERAPY; SAFETY; CRISPR-CAS9; PREVALENCE; PHENOTYPES;
D O I
10.3389/fgene.2021.794805
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
Inherited retinal diseases (IRDs) are a heterogenous group of orphan eye diseases that typically result from monogenic mutations and are considered attractive targets for gene-based therapeutics. Following the approval of an IRD gene replacement therapy for Leber's congenital amaurosis due to RPE65 mutations, there has been an intensive international research effort to identify the optimal gene therapy approaches for a range of IRDs and many are now undergoing clinical trials. In this review we explore therapeutic challenges posed by IRDs and review current and future approaches that may be applicable to different subsets of IRD mutations. Emphasis is placed on five distinct approaches to gene-based therapy that have potential to treat the full spectrum of IRDs: 1) gene replacement using adeno-associated virus (AAV) and nonviral delivery vectors, 2) genome editing via the CRISPR/Cas9 system, 3) RNA editing by endogenous and exogenous ADAR, 4) mRNA targeting with antisense oligonucleotides for gene knockdown and splicing modification, and 5) optogenetic approaches that aim to replace the function of native retinal photoreceptors by engineering other retinal cell types to become capable of phototransduction.
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页数:17
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