Emerging Concepts for RNA Therapeutics for Inherited Retinal Disease.
Emerging Concepts for RNA Therapeutics for Inherited Retinal Disease.
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DOI：
10.1007/978-3-030-27378-1_14
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发表时间：
2019
影响因子：
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通讯作者：
Spencer M. Moore;D. Skowronska-Krawczyk;Daniel L. Chao
中科院分区：
文献类型：
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作者：
Spencer M. Moore;D. Skowronska-Krawczyk;Daniel L. Chao
遗传性视网膜疾病（IRD）包括广泛的遗传性失明，具有显著的遗传异质性。在RNA水平上调节基因表达的治疗剂对于治疗IRD具有显著的前景。在这篇综述中，我们回顾了寡核苷酸治疗的分子基础，如核酶，RNA干扰（RNAi），反义寡核苷酸（阿索），CRISPRi/a，以及它们在IRD治疗中的应用。
Inherited retinal diseases (IRD) encompass a wide spectrum of hereditary blindness with significant genetic heterogeneity. Therapeutics regulating gene expression on an RNA level have significant promise for treating IRD. In this review, we review the molecular basis of oligonucleotide therapeutics such as ribozymes, RNA interference (RNAi), antisense oligonucleotides (ASO), CRISPRi/a, and their applications to treatments of IRD.