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box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Aspect\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-width: 1px; border-style: solid; border-bottom-color: rgb(204, 204, 204); border-right-color: rgb(204, 204, 204); word-break: break-all;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Small Nucleic Acid Drugs\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Small Molecule Drugs\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Antibody Drugs\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Target Specificity\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">High specificity to genetic sequences, targeting mRNA or non-coding RNAs.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Often bind active sites; risk of off-target effects.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">High specificity to protein epitopes.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Target Range\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Accesses &quot;undruggable&quot; targets like non-coding RNAs and inaccessible proteins.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Limited to targets with binding pockets.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Limited to extracellular or accessible proteins.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Mechanism of Action\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Modulates gene expression (e.g., RNAi, splicing modulation).\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Typically enzyme inhibition or receptor modulation.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Neutralizes antigens or triggers immune responses.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Development Time\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Rapid development due to direct sequence-based design.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Longer lead times for screening and optimization.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Time-intensive due to protein engineering.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Delivery Challenges\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Requires advanced delivery systems for stability and cellular uptake.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Generally easier delivery (oral, topical).\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Delivery can be challenging (injection only).\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Immunogenicity\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Low immunogenicity with chemical modifications.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Low to moderate depending on structure.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Potentially high, requires careful design.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Manufacturing\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Chemically synthesized, scalable, and reproducible.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Chemical synthesis, highly scalable.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Biologically produced, complex and costly.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003C\u002Ftbody>\u003C\u002Ftable>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Small nucleic acid drugs uniquely combine the specificity of antibody drugs with the versatility of small molecule drugs, offering innovative solutions for challenging therapeutic targets.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">GenCefe Biotech provides high-quality\u003Cspan style=\"font-family: Montserrat; color: rgb(79, 129, 189);\">&nbsp;\u003C\u002Fspan>\u003C\u002Fspan>\u003Ca href=\"https:\u002F\u002Fwww.gencefebio.com\u002FRNASynthesis\u002F20.html\" target=\"_blank\" style=\"text-decoration: underline; font-family: Montserrat; color: rgb(79, 129, 189);\">\u003Cspan style=\"font-family: Montserrat; color: rgb(79, 129, 189);\">siRNA\u003C\u002Fspan>\u003C\u002Fa>\u003Cspan style=\"font-family: Montserrat; color: rgb(79, 129, 189);\">,&nbsp;\u003C\u002Fspan>\u003Ca href=\"https:\u002F\u002Fwww.gencefebio.com\u002FRNASynthesis\u002F21.html\" target=\"_blank\" style=\"text-decoration: underline; font-family: Montserrat; color: rgb(79, 129, 189);\">\u003Cspan style=\"font-family: Montserrat; color: rgb(79, 129, 189);\">miRNA\u003C\u002Fspan>\u003C\u002Fa>\u003Cspan style=\"font-family: Montserrat; color: rgb(79, 129, 189);\">,&nbsp;\u003C\u002Fspan>\u003Ca href=\"https:\u002F\u002Fwww.gencefebio.com\u002FRNASynthesis\u002F22.html\" target=\"_blank\" style=\"text-decoration: underline; font-family: Montserrat; color: rgb(79, 129, 189);\">\u003Cspan style=\"font-family: Montserrat; color: rgb(79, 129, 189);\">sgRNA\u003C\u002Fspan>\u003C\u002Fa>\u003Cspan style=\"font-family: Montserrat; color: rgb(79, 129, 189);\">, \u003C\u002Fspan>\u003Cspan style=\"font-family: Montserrat;\">and&nbsp;\u003C\u002Fspan>\u003Ca href=\"https:\u002F\u002Fwww.gencefebio.com\u002FRNASynthesis\u002F10.html\" target=\"_blank\" style=\"text-decoration: underline; font-family: Montserrat; color: rgb(79, 129, 189);\">\u003Cspan style=\"font-family: Montserrat; color: rgb(79, 129, 189);\">custom RNA synthesis\u003C\u002Fspan>\u003C\u002Fa>\u003Cspan style=\"font-family: Montserrat;\">&nbsp;services. We can design and synthesize RNA Oligos of different lengths, different forms, and with various modifications according to customer’s needs, and also synthesize RNA sequences designed by customers. The RNA products we deliver are all purified by HPLC. ISO 9001-certified facilities and comprehensive quality control reports ensure the delivery of high-quality products.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>","2024-12-12 00:00:00","RNA Synthesis & Modifications—Unique characteristics of small nucleic acid drugs","Small nucleic acid drugs exhibit distinct features that set them apart from small molecule drugs and antibody-based therapies, such as high specificity to genetic sequences, rapid development, etc.","RNA Oligo, siRNA, miRNA, Antisense Oligo, Small nucleic acid drug, custom RNA synthesis","2025-04-28 17:02:56","76,75,74,73,72",[300,311,321,331,342],{"createBy":8,"createTime":8,"updateBy":8,"updateTime":8,"remark":8,"id":201,"categoryName":289,"title":301,"imgUrl":302,"content":303,"excerpt":90,"publishDate":304,"seoTitle":305,"seoDesc":306,"seoKeywords":307,"createDate":308,"sort":14,"relatedPostIds":309,"firstServiceProductId":61,"secondServiceProductIds":310,"relatedPost":8,"productService":8,"allSecondProductService":8,"twoSecondProductService":8},"What are the differences between miRNA and siRNA?","http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F30\u002F19_20260630154853A127.png","\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">\u003Cimg src=\"http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F30\u002F19_20260630154859A128.png\" title=\"miRNA and siRNA\" alt=\"miRNA and siRNA\" width=\"100%\"\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">MicroRNA (miRNA) and small interfering RNA (siRNA) are both short, non-coding RNA molecules involved in post-transcriptional gene regulation. Despite their similarities, they differ in origin, mechanism, and application.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Ctable cellspacing=\"0\">\u003Ctbody style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctr class=\"firstRow\" style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); text-align: center; border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cbr\u002F>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">miRNA\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">siRNA\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Origin\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-width: 1px; border-style: solid; border-bottom-color: rgb(204, 204, 204); border-right-color: rgb(204, 204, 204); word-break: break-all;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Endogenously encoded; processed from primary miRNA (pri-miRNA) to mature miRNA.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Often exogenous (synthetic or viral); processed from long double-stranded RNA (dsRNA).\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Structure\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Single-stranded with imperfectly paired hairpin structures.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Double-stranded RNA with perfect complementarity.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Mechanism of Action\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-width: 1px; border-style: solid; border-bottom-color: rgb(204, 204, 204); border-right-color: rgb(204, 204, 204); word-break: break-all;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Binds to partially complementary sequences in the 3&#39; UTR of mRNA, leading to translational repression or degradation.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Binds to perfectly complementary sequences, inducing target mRNA cleavage and degradation.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Target Specificity\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Broad target range; regulates multiple genes due to partial complementarity.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Highly specific; targets a single mRNA due to perfect base pairing.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Biological Function\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Regulates endogenous gene expression; involved in development, cell differentiation, and stress responses.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Acts as a defense mechanism against viruses and transposable elements.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Applications\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Biomarkers for disease diagnostics and prognostics; potential for modulating multiple pathways in therapy.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd valign=\"top\" style=\"padding: 3px 5px; margin: 0px; box-sizing: border-box; border-bottom: 1px solid rgb(204, 204, 204); border-right: 1px solid rgb(204, 204, 204); border-top-width: 1px; border-left-width: 1px; border-top-style: solid; border-left-style: solid;\">\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Research tools for gene silencing; therapeutics for precise gene targeting (e.g., ONPATTRO).\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003C\u002Ftbody>\u003C\u002Ftable>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">While both miRNA and siRNA are crucial players in RNA interference, they differ significantly in their origin, targeting mechanisms, and functional roles. miRNAs are endogenous regulators with broader, less specific effects, whereas siRNAs are more specific tools for precise gene silencing, making them complementary in research and therapeutic applications.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>","2025-02-27 00:00:00","RNA Synthesis & Modifications—Differences between miRNA and siRNA","miRNAs are endogenous regulators with broader, less specific effects, whereas siRNAs are more specific tools for precise gene silencing, making them complementary in research and therapeutic applications."," RNA Oligo, miRNA, microRNA, siRNA, RNAi, RNA interference, Small nucleic acid drug, custom RNA synthesis","2025-04-30 16:49:36","61,60,59,58","13,56",{"createBy":8,"createTime":8,"updateBy":8,"updateTime":8,"remark":8,"id":204,"categoryName":289,"title":312,"imgUrl":313,"content":314,"excerpt":90,"publishDate":304,"seoTitle":315,"seoDesc":316,"seoKeywords":317,"createDate":318,"sort":17,"relatedPostIds":319,"firstServiceProductId":61,"secondServiceProductIds":320,"relatedPost":8,"productService":8,"allSecondProductService":8,"twoSecondProductService":8},"What is the current progress in antisense oligonucleotide (ASO) drug development? ","http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F30\u002F20_20260630155023A129.png","\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">\u003Cimg src=\"http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F30\u002F20_20260630155029A130.png\" title=\"antisense oligonucleotide (ASO) drug development\" alt=\"antisense oligonucleotide (ASO) drug development\" width=\"100%\"\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Antisense oligonucleotide (ASO) drugs have advanced significantly, establishing themselves as a versatile and effective platform for treating genetic and other complex diseases. Key progress includes the following:&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">1. Approved ASO Therapies&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Several ASO drugs have been approved for clinical use, addressing a wide range of conditions:&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp;&nbsp;&nbsp;●&nbsp; &nbsp; &nbsp; Nusinersen (Spinraza):&nbsp;The first FDA-approved ASO for spinal muscular atrophy (SMA), developed by Ionis Pharmaceuticals and Biogen. It targets the SMN2 gene to enhance functional SMN protein production.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp;&nbsp;&nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Mipomersen (Kynamro):&nbsp;Used to treat familial hypercholesterolemia by reducing apolipoprotein B (ApoB) production.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp;&nbsp;&nbsp;●&nbsp; &nbsp; &nbsp; Eteplirsen (Exondys 51):&nbsp;Targets exon skipping in the dystrophin gene for Duchenne muscular dystrophy (DMD).&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">2. Expanding Disease Applications&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Neurological Diseases:&nbsp;ASOs have shown promise in treating Huntington’s disease (e.g., Tominersen) and amyotrophic lateral sclerosis (ALS). These drugs are designed to reduce harmful protein accumulation or correct splicing defects.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Oncology:&nbsp;ASOs are being investigated to silence oncogenes or re-sensitize tumors to chemotherapy.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Metabolic Disorders:&nbsp;Targeting liver-specific pathways, ASOs like inclisiran reduce low-density lipoprotein (LDL) cholesterol.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">3. Innovations in Chemistry&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Chemical modifications have improved ASO stability, delivery, and efficacy:&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Phosphorothioate Backbones:&nbsp;Enhance stability and resistance to nucleases.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; 2&#39;-O-Methyl (2&#39;-OMe) and 2&#39;-Methoxyethyl (2&#39;-MOE):&nbsp;Increase binding affinity and reduce toxicity.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Gapmers:&nbsp;ASOs designed to activate RNase H-mediated cleavage, improving potency.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">4. Delivery Advancements&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Targeted Delivery:&nbsp;Liver-specific delivery through conjugates like N-acetylgalactosamine (GalNAc) has reduced off-target effects and improved safety profiles.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Intrathecal Administration:&nbsp;Direct delivery to the central nervous system (CNS) for neurological diseases, bypassing the blood-brain barrier.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">5. Ongoing Clinical Trials&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Dozens of ASO drugs are in clinical development, targeting diseases such as Alzheimer’s, cancer, and rare genetic conditions. Emerging therapies include RNA-targeting ASOs for exon skipping, splicing correction, and gene silencing.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">ASO drug development has achieved remarkable progress, with approved therapies addressing rare genetic diseases and expanding into more prevalent conditions like cancer and neurological disorders. Advances in chemistry, delivery systems, and disease-specific targeting are driving the growth of ASOs as a transformative therapeutic platform.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>","RNA Synthesis & Modifications—Progress in antisense oligonucleotide (ASO) drug development"," ASO drug development has achieved remarkable progress, with approved therapies addressing rare genetic diseases and expanding into more prevalent conditions like cancer and neurological disorders.","RNA Oligo, ASO, antisense oligo, Small nucleic acid drug, custom RNA synthesis","2025-04-30 16:50:17","67,66,65,64,63","13,60",{"createBy":8,"createTime":8,"updateBy":8,"updateTime":8,"remark":8,"id":175,"categoryName":289,"title":322,"imgUrl":323,"content":324,"excerpt":90,"publishDate":325,"seoTitle":326,"seoDesc":327,"seoKeywords":328,"createDate":329,"sort":268,"relatedPostIds":330,"firstServiceProductId":61,"secondServiceProductIds":320,"relatedPost":8,"productService":8,"allSecondProductService":8,"twoSecondProductService":8},"What are the differences between ASO and siRNA?","http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2025\u002F08\u002F08\u002F06-min_20250808154306A026.jpg","\u003Cp style=\"text-align:center\">\u003Cspan style=\"font-family: Montserrat;\">\u003Cimg src=\"http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F23\u002F1_20260623151151A001.png\" width=\"100%\" title=\"differences between ASO and siRNA\" alt=\"differences between ASO and siRNA\"\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are both tools for modulating gene expression, but they differ in structure, mechanism, delivery, and applications. ASOs are single-stranded molecules designed to bind target RNA and alter its function, while siRNAs are double-stranded molecules that trigger RNA interference (RNAi) to degrade target mRNA. Below is a detailed comparison.\u003C\u002Fspan>\u003C\u002Fp>\u003Ctable cellspacing=\"0\" border=\"1px\" width=\"100%\">\u003Ctbody style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctr class=\"firstRow\" style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd>\u003Cbr\u002F>\u003C\u002Ftd>\u003Ctd align=\"center\">\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">ASO (Antisense Oligonucleotide)\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd align=\"center\">\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">siRNA (Small Interfering RNA)\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd valign=\"top\" style=\"white-space: nowrap\">\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">Structure\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Single-stranded DNA or RNA molecule.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Double-stranded RNA molecule with a guide strand and a passenger strand.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd>\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">Mechanism of Action\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Binds to target RNA through complementary base pairing, modulating gene expression via RNase H activation or steric hindrance.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Binds perfectly complementary RNA, inducing target mRNA cleavage via the RISC complex.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd>\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">Delivery\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Delivered as single molecules; typically conjugated with ligands like GalNAc for targeted delivery.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Delivered as duplex RNA; often formulated with nanoparticles or lipid-based carriers.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd>\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">Target Specificity\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Can target both pre-mRNA (splicing modulation) and mature mRNA.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Targets mature mRNA only due to its mechanism requiring perfect complementarity.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd>\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">Applications\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Splicing modulation, gene knockdown, and disease-specific RNA regulation.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Gene silencing through degradation of specific mRNA.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd>\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">Stability\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Chemically modified for increased nuclease resistance and longer half-life.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Less stable due to inherent susceptibility to nuclease degradation.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd>\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">Tissue Targeting\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Can be targeted to specific tissues, such as liver or CNS, via chemical conjugates.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Typically relies on delivery systems for effective tissue targeting.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003Ctr style=\"padding: 0px; margin: 0px; box-sizing: border-box;\">\u003Ctd>\u003Cp>\u003Cstrong>\u003Cspan style=\"font-family: Montserrat;\">Examples\u003C\u002Fspan>\u003C\u002Fstrong>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Nusinersen, Mipomersen, Eteplirsen.\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003Ctd>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">ONPATTRO (Patisiran), GIVLAARI (Givosiran).\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Ftd>\u003C\u002Ftr>\u003C\u002Ftbody>\u003C\u002Ftable>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">ASOs and siRNAs are complementary tools in therapeutic development, each with unique advantages. ASOs offer versatility in targeting both pre-mRNA and mature RNA, enabling broader applications like splicing modulation. In contrast, siRNAs are highly effective for precise mRNA degradation through RNAi. Advances in chemistry and delivery technologies have expanded their therapeutic potential, making both ASOs and siRNAs valuable in addressing a variety of diseases.\u003C\u002Fspan>\u003Cbr\u002F>\u003C\u002Fp>","2025-03-04 00:00:00","RNA Synthesis & Modifications—Differences between ASO and siRNA","ASOs offer versatility in targeting both pre-mRNA and mature RNA, enabling broader applications like splicing modulation. In contrast, siRNAs are highly effective for precise mRNA degradation through RNAi.","RNA Oligo, ASO, antisense oligo, siRNA, RNAi, RNA interference, Small nucleic acid drug, custom RNA synthesi","2025-04-30 16:51:04","75,73,72,58,54",{"createBy":8,"createTime":8,"updateBy":8,"updateTime":8,"remark":8,"id":332,"categoryName":289,"title":333,"imgUrl":334,"content":335,"excerpt":90,"publishDate":336,"seoTitle":337,"seoDesc":338,"seoKeywords":339,"createDate":340,"sort":101,"relatedPostIds":341,"firstServiceProductId":61,"secondServiceProductIds":320,"relatedPost":8,"productService":8,"allSecondProductService":8,"twoSecondProductService":8},75,"What are the ASO pipelines currently in clinical stages? ","http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F30\u002F21_20260630155158A131.png","\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">\u003Cimg src=\"http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F30\u002F21_20260630155203A132.png\" title=\"ASO pipelines\" alt=\"ASO pipelines\" width=\"100%\"\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Antisense oligonucleotide (ASO) drugs have shown significant progress in clinical development, targeting a wide range of diseases, from rare genetic disorders to more common conditions. Since the approval of the first ASO drug, Fomivirsen, in 1998, the pipeline has expanded substantially. Here are some key ASO drugs currently in clinical stages:&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Approved ASO Drugs&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Nusinersen (Spinraza):&nbsp;Approved in 2016 by the FDA for treating spinal muscular atrophy (SMA). It enhances the production of functional SMN protein.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Eteplirsen:&nbsp;Approved for Duchenne muscular dystrophy (DMD), it promotes exon skipping to restore functional dystrophin protein.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Inotersen:&nbsp;Approved for hereditary transthyretin-mediated amyloidosis (hATTR), it reduces transthyretin protein levels to slow disease progression.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">ASO Drugs in Clinical Trials&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Tominersen:&nbsp;Designed for Huntington’s disease, it reduces mutant huntingtin protein levels. Currently in Phase II clinical trials.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; ON363:&nbsp;Targets FUS-related amyotrophic lateral sclerosis (ALS) by decreasing FUS protein production. It has reached Phase III clinical trials.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; ALG-020572:&nbsp;A novel ASO for chronic hepatitis B (CHB) treatment, currently in Phase I trials.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; IONIS-TTR-LRx:&nbsp;A liver-targeting ASO for hATTR amyloidosis, improving delivery efficiency with GalNAc conjugation.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Global and Regional Progress\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">ASO development is advancing globally, with contributions from leading biotechnology companies and institutions. In China, domestic enterprises such as Hengrui Medicine are developing ASO drugs, with 16 oligonucleotide-based candidates approved for clinical studies, marking a significant milestone in local innovation.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">The ASO clinical pipeline has grown to address diverse diseases, including neurodegenerative disorders, rare genetic diseases, and viral infections. Advances in chemical modifications, delivery systems, and disease-specific targeting continue to drive the evolution of ASO therapeutics. These developments promise to bring more ASO drugs to market, benefiting patients worldwide.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>","2025-03-06 00:00:00","RNA Synthesis & Modifications—ASO pipelines currently in clinical stages","The ASO clinical pipeline has grown to address diverse diseases, including neurodegenerative disorders, rare genetic diseases, and viral infections.","RNA Oligo, ASO, antisense oligo, RNAi, RNA interference, Small nucleic acid drug, custom RNA synthesis","2025-04-30 16:51:38","71,67,64,60",{"createBy":8,"createTime":8,"updateBy":8,"updateTime":8,"remark":8,"id":343,"categoryName":289,"title":344,"imgUrl":345,"content":346,"excerpt":90,"publishDate":347,"seoTitle":348,"seoDesc":349,"seoKeywords":350,"createDate":351,"sort":211,"relatedPostIds":352,"firstServiceProductId":61,"secondServiceProductIds":353,"relatedPost":8,"productService":8,"allSecondProductService":8,"twoSecondProductService":8},76,"What is the current progress in sgRNA drug research?","http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F30\u002F22_20260630155259A133.png","\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">\u003Cimg src=\"http:\u002F\u002Fmanage.gencefebio.com\u002Fprofile\u002Fupload\u002F2026\u002F06\u002F30\u002F22_20260630155306A134.png\" title=\"sgRNA drug research\" alt=\"sgRNA drug research\" width=\"100%\"\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">Single-guide RNA (sgRNA) has emerged as a pivotal tool in CRISPR-based genome editing, facilitating precise and programmable modifications of DNA or RNA. As part of CRISPR-Cas systems, sgRNA plays a central role in directing Cas proteins to specific genomic loci. Its therapeutic applications have progressed significantly, with notable advancements in treating genetic diseases, cancers, and infectious diseases.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">1. Therapeutic Applications&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Genetic Disorders:&nbsp;sgRNA-guided CRISPR systems are used to correct pathogenic mutations in diseases such as sickle cell anemia, β-thalassemia, and cystic fibrosis. Ongoing clinical trials aim to demonstrate the safety and efficacy of these approaches.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Oncology:&nbsp;sgRNA is being explored to target oncogenes, enhance immune checkpoint inhibition, or engineer CAR-T cells for improved anti-tumor activity.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Infectious Diseases:&nbsp;CRISPR-sgRNA systems are applied to combat viral infections, such as HIV and hepatitis B, by targeting viral genomes for degradation.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">2. Advancements in sgRNA Design&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Enhanced Specificity:&nbsp;Improved sgRNA algorithms and chemical modifications have reduced off-target effects, ensuring higher precision in genome editing.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Multiplexing:&nbsp;Advanced sgRNA strategies allow simultaneous targeting of multiple genes, broadening therapeutic potential.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Base Editing and Prime Editing:&nbsp;sgRNAs now facilitate precise base conversions and targeted DNA insertions without introducing double-strand breaks, minimizing unintended consequences.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">3. Delivery Innovations&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Lipid Nanoparticles (LNPs):&nbsp;Widely used for delivering sgRNA-Cas complexes in vivo, particularly for liver-targeted therapies.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Adeno-associated Viruses (AAVs):&nbsp;Commonly employed for delivering sgRNA and Cas components to specific tissues, with promising results in preclinical studies.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Ribonucleoprotein (RNP) Complexes:&nbsp;Direct delivery of sgRNA pre-complexed with Cas proteins has shown improved editing efficiency and reduced immunogenicity.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">4. Clinical Progress&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Ex Vivo Editing:&nbsp;Several clinical trials use sgRNA-directed CRISPR to edit patient-derived cells, such as hematopoietic stem cells, before reintroducing them to the patient.&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; In Vivo Editing:&nbsp;Companies like Intellia Therapeutics are conducting trials using sgRNA-based CRISPR systems for in vivo genome editing, targeting conditions like transthyretin amyloidosis (ATTR).&nbsp;\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">&nbsp; &nbsp; &nbsp;●&nbsp; &nbsp; &nbsp; Ongoing Trials:&nbsp;Trials for conditions including Leber congenital amaurosis (LCA), sickle cell anemia, and β-thalassemia are demonstrating encouraging preliminary results.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>\u003Cp>\u003Cspan style=\"font-family: Montserrat;\">sgRNA-based therapeutics have revolutionized genome editing, offering unprecedented precision and versatility. Advances in sgRNA design, delivery, and clinical translation are driving progress, with several therapies nearing commercialization. Continued innovation in this field holds the promise of addressing previously untreatable genetic and complex diseases.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp>\u003Cbr\u002F>\u003C\u002Fp>","2025-03-11 00:00:00","RNA Synthesis & Modifications—Progress in sgRNA drug research","Advances in sgRNA design, delivery, and clinical translation are driving progress, with several therapies nearing commercialization. Continued innovation in this field holds the promise of addressing previously untreatable genetic and complex diseases.","RNA Oligo, sgRNA, guide RNA, gene editing, CRISPR, Small nucleic acid drug, custom RNA synthesis","2025-04-30 16:52:26","73,69,65,63,60","13,58"]