RNA-Based Therapeutic Strategies for Inherited Retinal Dystrophies

被引：8

作者：

Garanto, Alejandro ^{[1
,2
]}

机构：

[1] Radboud Univ Nijmegen, Med Ctr, Dept Human Genet, Nijmegen, Netherlands

[2] Radboud Univ Nijmegen, Med Ctr, Donders Inst Brain Cognit & Behav, Nijmegen, Netherlands

来源：

RETINAL DEGENERATIVE DISEASES: MECHANISMS AND EXPERIMENTAL THERAPY | 2019年 / 1185卷

关键词：

RNA-based therapies; RNA editing; Antisense oligonucleotides; Trans-splicing; U1snRNA; siRNA; Cas13; ANTISENSE OLIGONUCLEOTIDE; MESSENGER-RNA; U1; SNRNA; FOMIVIRSEN; THERAPIES; RHODOPSIN; MUTATION; RESCUE; DESIGN; SAFETY;

D O I：

10.1007/978-3-030-27378-1_12

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Inherited retinal dystrophies (IRDs) are genetic diseases affecting 1 in every 3000 individuals worldwide. Nowadays, more than 250 genes have been associated with different forms of IRD. In the last decade, it has been shown that gene therapy is a promising approach to correct the genetic defects underlying IRD. In fact, voretigene neparvovec-rzyl (Luxturna (TM)), the first commercialized gene therapy drug to treat RPE65-associated Leber congenital amaurosis, has opened new venues. However, IRDs are highly heterogeneous at genetic level making the design of novel strategies complicated. Unfortunately, the size of several frequently mutated genes is not suitable for the approved conventional therapeutic viral vectors; therefore, there is an urgent need for the development of alternatives, such as those targeting the pre-mRNA. In this mini-review, the potential of RNA-based strategies for IRDs is discussed.

引用

页码：71 / 77

页数：7

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