摘要
小活化RNA(saRNA)是小的双链RNA,其可以通过在转录和表观遗传水平上靶向基因启动子中的选定序列来介导靶特异性基因表达。这种基因操作现象被称为RNA激活(RNAa),与RNA干扰相比,它开辟了基于RNA的基因治疗的新途径。虽然由saRNA介导的RNAa的确切分子机制仍然仍然模糊,但一些研究提供了可能的解释它。此外,越来越多的证据表明,saRNA不仅提供了研究基因功能和操纵转录活性的新方法,而且还为临床治疗各种疾病,特别是癌症提供了巨大的潜力。癌症相关基因可被saRNA上调,以调节细胞周期和增殖,诱导细胞衰老和凋亡,抑制癌细胞侵袭和迁移,并逆转化疗耐药性。在此,我们总结了saRNA在上调特定基因表达方面的已知机制,并着重于saRNA在基因治疗中的潜在应用。此外,本综述还涉及对传递saRNA的机制和挑战的一些担忧。需要进一步说明saRNA的精确机制,并且期望开发用于saRNA的一些新型递送系统用于临床应用
关键词: 小活化RNA,RNA激活,基于RNA的基因治疗,基因治疗,癌症治疗,药物输送系统
Current Gene Therapy
Title:Current Advances in Small Activating RNAs for Gene Therapy: Principles, Applications and Challenges
Volume: 18 Issue: 3
关键词: 小活化RNA,RNA激活,基于RNA的基因治疗,基因治疗,癌症治疗,药物输送系统
摘要: Small activating RNAs (saRNAs) are small double-stranded RNAs that could mediate the target-specific gene expression by targeting selected sequences in gene promoters at both the transcriptional and epigenetic levels. This phenomenon of gene manipulation is known as RNA activation (RNAa), which opens up a new pathway for RNA-based gene therapeutics in contrast to RNA interference. Although the exact molecular mechanism of RNAa mediated by saRNAs still remains foggy, some studies have provided the possible ones to explain it. Furthermore, mounting evidence exhibit that saRNAs not only provide a new approach to study gene function and manipulate transcriptional activity, but also promise a great potential for clinical therapy against various diseases, especially cancer. Cancer-associated genes could be up-regulated by saRNAs to modulate cell cycle and proliferation, induce cell senescence and apoptosis, inhibit cancer cell invasion and migration, and reverse chemotherapy resistance. Herein, we summarize the known mechanisms of saRNAs on up-regulating specific gene expression and focus on the potential applications of saRNAs in gene therapy. In addition, some concerns about mechanisms and challenges for delivery of saRNAs are involved in this review. The precise mechanisms of saRNAs need to be further illustrated and some novel delivery systems for saRNAs are expected to be developed for clinical applications.
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Current Advances in Small Activating RNAs for Gene Therapy: Principles, Applications and Challenges, Current Gene Therapy 2018; 18 (3) . https://dx.doi.org/10.2174/1566523218666180619155018
DOI https://dx.doi.org/10.2174/1566523218666180619155018 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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