RNA therapeutics for retinal diseases.
RNA therapeutics for retinal diseases.
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DOI：
10.1080/14712598.2021.1856365
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发表时间：
2021-05
影响因子：
4.6
通讯作者：
Ciulla T
中科院分区：
文献类型：
--
作者：
Gemayel MC;Bhatwadekar AD;Ciulla T
在视网膜中，非编码RNA（ncRNA）在调节细胞凋亡、炎症反应、视觉感知和光转导中起着不可或缺的作用，在疾病状态下报告了改变的水平。MicroRNA（miRNA）是一类非编码RNA，通过与靶信使RNA（mRNA）的互补结合位点结合，产生翻译抑制，从而调控转录后基因的表达。小干扰RNA（siRNA）是调节基因表达的双链RNA（dsRNA），通过称为RNA干扰（RNAi）的过程导致基因的选择性沉默。另一种形式的RNAi涉及短发夹RNA（shRNA）。在年龄相关性黄斑变性（AMD）和糖尿病视网膜病变（DR）中，miRNA参与了血管生成、氧化应激、免疫反应和炎症的调节。许多正在开发的基于RNA的疗法可以方便地通过玻璃体内给药，具有全视网膜效应的潜力。这些RNA治疗剂中的大多数是合成的ncRNA，并且有望用于治疗AMD、DR和遗传性视网膜疾病（IRD）。这些基于RNA的疗法包括具有高特异性的siRNA疗法、用于“敲低”常染色体显性毒性获得的功能突变基因的shRNA、可以恢复剪接缺陷的反义寡核苷酸（ASO）和用于增加具有过早终止密码子的基因的全长蛋白质表达的翻译通读诱导药物（TRIDS）。
In the retina, noncoding RNA (ncRNA) plays an integral role in regulating apoptosis, inflammatory responses, visual perception, and photo-transduction, with altered levels reported in diseased states. MicroRNA (miRNA), a class of ncRNA, regulates post-transcription gene expression through the binding of complementary binding sites of target messenger RNA (mRNA) with resulting translational repression. Small interfering RNA (siRNA) are double-stranded RNA (dsRNA) that regulate gene expression, leading to selective silencing of genes through a process called RNA interference (RNAi). Another form of RNAi involves short hairpin RNA (shRNA). In age-related macular degeneration (AMD) and in diabetic retinopathy (DR), miRNA has been implicated in the regulation of angiogenesis, oxidative stress, immune response, and inflammation. Many RNA-based therapies in development are conveniently administered intravitreally, with potential for panretinal effect. The majority of these RNA therapeutics are synthetic ncRNA’s and hold promise for the treatment of AMD, DR and inherited retinal diseases (IRDs). These RNA-based therapies include siRNA therapy with its high specificity, shRNA to “knock down” autosomal dominant toxic gain of function mutated genes, antisense oligonucleotides (ASOs), which can restore splicing defects, and translational read-through inducing drugs (TRIDS) to increase expression of full-length protein from genes with premature stop codons.
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