共 74 条
RNA therapeutics for retinal diseases
被引:25
作者:
Gemayel, Michael C.
[1
]
Bhatwadekar, Ashay D.
[1
]
Ciulla, Thomas
[1
,2
,3
]
机构:
[1] Indiana Univ, Dept Ophthalmol, Eugene & Marilyn Glick Eye Inst, Indianapolis, IN 46202 USA
[2] Clearside Biomed Inc, Preclin & Clin Dev, Alpharetta, GA USA
[3] Midwest Eye Inst, Indianapolis, IN USA
基金:
美国国家卫生研究院;
关键词:
Antisense oligonucleotides;
inherited retinal disease;
microRNA;
noncoding RNA;
RNA therapeutics;
short hairpin RNA;
small-interfering RNA;
translational read-through inducing drugs;
TRANSLATIONAL READ-THROUGH;
CHOROIDAL NEOVASCULARIZATION;
ANTISENSE OLIGONUCLEOTIDES;
CYTOMEGALOVIRUS RETINITIS;
DIABETIC-RETINOPATHY;
MACULAR DEGENERATION;
DOWN-REGULATION;
MICRORNAS;
DELIVERY;
MECHANISMS;
D O I:
10.1080/14712598.2021.1856365
中图分类号:
Q81 [生物工程学(生物技术)];
Q93 [微生物学];
学科分类号:
071005 ;
0836 ;
090102 ;
100705 ;
摘要:
Introduction 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. Areas covered MicroRNA (miRNA), a class of ncRNA, regulates post-transcription gene expression through the binding of complementary sites of target messenger RNA (mRNA) with resulting translational repression. Small-interfering RNA (siRNA) is a double-stranded RNA (dsRNA) that regulates 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 diabetic retinopathy (DR), miRNA has been implicated in the regulation of angiogenesis, oxidative stress, immune response, and inflammation. Expert opinion Many RNA-based therapies in development are conveniently administered intravitreally, with the potential for pan-retinal 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.
引用
收藏
页码:603 / 613
页数:11
相关论文