siRNA是治疗糖尿病并发症的有前途的工具

Q2 Pharmacology, Toxicology and Pharmaceutics OpenNano Pub Date : 2023-09-01 DOI:10.1016/j.onano.2023.100174
Moqbel Ali Moqbel Redhwan , Hariprasad M.G , Suman Samaddar , Sumaia Abdulbari Ahmed Ali Hard
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摘要

RNA干扰(RNAi)是一种自然发生的基因调控过程,已被利用来沉默各种细胞类型中的特定基因,包括与糖尿病并发症有关的基因。小干扰RNA (Small interfering RNA, siRNA)是一种激活RNAi并针对特定基因进行降解的RNA分子。最近的研究表明,siRNA有望成为治疗糖尿病并发症的工具,包括糖尿病神经病变、视网膜病变和肾病。在临床前研究中,siRNA已被证明能有效靶向与这些并发症相关的基因,从而改善临床结果。siRNA治疗的一个潜在优势是它能够选择性地靶向特定基因而不破坏内源性mRNA通路,从而降低脱靶效应的风险。此外,单剂量siRNA有可能提供持久的效果,这可能导致减少治疗频率和提高患者的依从性。虽然有希望的临床前结果已经出现,但在siRNA可以用于临床实践之前,仍然需要解决几个挑战。其中包括递送问题,因为siRNA分子在血液中迅速降解,没有帮助就不能穿过细胞膜。尽管存在这些挑战,siRNA作为治疗糖尿病并发症的工具的潜力是令人兴奋的,需要进一步的研究来确定其在临床试验中的安全性和有效性。随着不断的研究和完善,siRNA有可能成为治疗糖尿病并发症的重要治疗工具,改善患者的预后和生活质量。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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siRNA a promising tool for diabetes complications

RNA interference (RNAi) is a naturally occurring process of gene regulation that has been harnessed to silence specific genes in various cell types, including those involved in diabetes complications. Small interfering RNA (siRNA) is an RNA molecule that activates RNAi and targets specific genes for degradation. Recent research has demonstrated that siRNA holds promise as a tool for treating diabetes complications, including diabetic neuropathy, retinopathy, and nephropathy. In preclinical studies, siRNA has been shown to effectively target genes involved in these complications, resulting in improved clinical outcomes. One potential advantage of siRNA therapy is its ability to selectively target specific genes without disrupting endogenous mRNA pathways, which reduces the risk of off-target effects. Additionally, siRNA has the potential to provide long-lasting effects with a single dose, which could result in reduced treatment frequency and improved patient compliance. While promising preclinical results have been, several challenges still need to be addressed before siRNA can be used in clinical practice. These include delivery issues, as siRNA molecules rapidly degrade in the bloodstream and cannot cross cell membranes without assistance. Despite these challenges, the potential of siRNA as a tool for treating diabetes complications is exciting, and further research is needed to determine its safety and efficacy in clinical trials. With continued investigation and refinement, siRNA has the potential to become an important therapeutic tool for the treatment of diabetes complications, improving patient outcomes and quality of life.

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来源期刊
OpenNano
OpenNano Medicine-Pharmacology (medical)
CiteScore
4.10
自引率
0.00%
发文量
63
审稿时长
50 days
期刊介绍: OpenNano is an internationally peer-reviewed and open access journal publishing high-quality review articles and original research papers on the burgeoning area of nanopharmaceutics and nanosized delivery systems for drugs, genes, and imaging agents. The Journal publishes basic, translational and clinical research as well as methodological papers and aims to bring together chemists, biochemists, cell biologists, material scientists, pharmaceutical scientists, pharmacologists, clinicians and all others working in this exciting and challenging area.
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