Cancer Institute, The Affiliated Hospital of Qingdao University, Qingdao University, Qingdao Cancer Institute, Qingdao, Shandong, 266000, China.
Department of Liver Disease Center, The Affiliated Hospital of Qingdao University, Qingdao, Shandong, 266000, China.
Int J Biol Sci. 2023 May 29;19(9):2879-2896. doi: 10.7150/ijbs.84994. eCollection 2023.
Cholesterol levels are an initiating risk factor for atherosclerosis. Many genes play a central role in cholesterol synthesis, including HMGCR, SQLE, HMGCS1, FDFT1, LSS, MVK, PMK, MVD, FDPS, CYP51, TM7SF2, LBR, MSMO1, NSDHL, HSD17B7, DHCR24, EBP, SC5D, DHCR7, IDI1/2. Especially, HMGCR, SQLE, FDFT1, LSS, FDPS, CYP51, and EBP are promising therapeutic targets for drug development due to many drugs have been approved and entered into clinical research by targeting these genes. However, new targets and drugs still need to be discovered. Interestingly, many small nucleic acid drugs and vaccines were approved for the market, including Inclisiran, Patisiran, Inotersen, Givosiran, Lumasiran, Nusinersen, Volanesorsen, Eteplirsen, Golodirsen, Viltolarsen, Casimersen, Elasomeran, Tozinameran. However, these agents are all linear RNA agents. Circular RNAs (circRNAs) may have longer half-lives, higher stability, lower immunogenicity, lower production costs, and higher delivery efficiency than these agents due to their covalently closed structures. CircRNA agents are developed by several companies, including Orna Therapeutics, Laronde, and CirCode, Therorna. Many studies have shown that circRNAs regulate cholesterol synthesis by regulating HMGCR, SQLE, HMGCS1, ACS, YWHAG, PTEN, DHCR24, SREBP-2, and PMK expression. MiRNAs are essential for circRNA-mediated cholesterol biosynthesis. Notable, the phase II trial for inhibiting miR-122 with nucleic acid drugs has been completed. Suppressing HMGCR, SQLE, and miR-122 with circRNA_ABCA1, circ-PRKCH, circEZH2, circRNA-SCAP, and circFOXO3 are the promising therapeutic target for drug development, specifically the circFOXO3. This review focuses on the role and mechanism of the circRNA/miRNA axis in cholesterol synthesis in the hope of providing knowledge to identify new targets.
胆固醇水平是动脉粥样硬化的起始风险因素。许多基因在胆固醇合成中发挥核心作用,包括 HMGCR、SQLE、HMGCS1、FDFT1、LSS、MVK、PMK、MVD、FDPS、CYP51、TM7SF2、LBR、MSMO1、NSDHL、HSD17B7、DHCR24、EBP、SC5D、DHCR7、IDI1/2。特别是,HMGCR、SQLE、FDFT1、LSS、FDPS、CYP51 和 EBP 是很有前途的药物开发治疗靶点,因为许多药物已经被批准并进入临床研究,这些药物针对这些基因。然而,仍然需要发现新的靶点和药物。有趣的是,许多小核酸药物和疫苗已获准上市,包括 Inclisiran、Patisiran、Inotersen、Givosiran、Lumasiran、Nusinersen、Volanesorsen、Eteplirsen、Golodirsen、Viltolarsen、Casimersen、Elasomeran、Tozinameran。然而,这些药物都是线性 RNA 药物。由于其共价闭合结构,环状 RNA (circRNA) 可能具有更长的半衰期、更高的稳定性、更低的免疫原性、更低的生产成本和更高的递送效率。circRNA 药物由几家公司开发,包括 Orna Therapeutics、Laronde 和 CirCode、Therorna。许多研究表明,circRNA 通过调节 HMGCR、SQLE、HMGCS1、ACS、YWHAG、PTEN、DHCR24、SREBP-2 和 PMK 的表达来调节胆固醇合成。miRNA 对 circRNA 介导的胆固醇生物合成至关重要。值得注意的是,用核酸药物抑制 miR-122 的 II 期临床试验已经完成。用 circRNA_ABCA1、circ-PRKCH、circEZH2、circRNA-SCAP 和 circFOXO3 抑制 HMGCR、SQLE 和 miR-122 是药物开发的有前途的治疗靶点,特别是 circFOXO3。本综述重点介绍 circRNA/miRNA 轴在胆固醇合成中的作用和机制,希望为识别新靶点提供知识。
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