图3. Strategy for obtaining high-affinity chemically modified mannose–siRNA conjugate[2]除了上述问题之外，siRNA药物还需要具备良好的靶向性，即能够在体内特异性地定位到病变部位，并在那里释放出足够的剂量以产生治疗效果。然而，当前的递送技术往往难以精确控制siRNA的分布模式，导致药物可能分散到非目标区域，增加...
对于非肝脏组织而言,siRNA药物的递送面临着显著的技术挑战。尤其是当涉及到siRNA从内吞体逃逸到细胞质的过程时,这一过程在非肝细胞中显得尤为困难。这主要是因为大多数非肝细胞表面受体的数量远低于肝细胞上的唾液酸糖蛋白受体(ASGPR),后者能够帮助Gal...
然而,对于许多非肝细胞而言,这一过程效率极低,限制了siRNA的效果。 图3. Strategy for obtaining high-affinity chemically modified mannose–siRNA conjugate[2] 除了上述问题之外,siRNA药物还需要具备良好的靶向性,即能够在体内特异性地定位到病变部位,并在那里释放出足够的剂量以产生治疗效果。然而,当前的递送技术往...
56. Jeong JH, Mok HJ, Oh Y, Park TG. siRNA conjugate delivery systems. Bioconjug Chem. 2009;20:5–14. 57. Jeong JH, Kim SW, Park TG. Novel intracellular delivery system of antisense oligonucleotide by self-assembled hybrid micelles composed of DNA/P...
RNAi therapeutics utilizing GalNAc conjugate technology are administered subcutaneously. Our medicines GIVLAARI® (givosiran), OXLUMO® (lumasiran),AMVUTTRA® (vutrisiran), and Leqvio® (inclisiran)* utilize GalNAc conjugate delivery.
Figure1: A. Structure of PEG-siRNA conjugate; B. Formation of PEC micelles from the interactions between PEG-siRNA conjugate and a polycation (Jeong, J. H. 2009). References: Jeong, J. H.et al. siRNA Conjugate Delivery Systems.Bioconjugate Chemistry. 2009; 20(1):5–14. ...
[3] Mihaila, R. et al. Mathematical Modeling: A Tool for Optimization of Lipid Nanoparticle-Mediated Delivery of siRNA. Mol. Ther. - Nucleic Acids 7, 246–255 (2017). [4] Witzigmann, D. et al. Variable asialoglycoprotein receptor 1 expression in liver disease: Implications for therapeut...
siRNA-Nanoparticle Conjugate in Gene Silencing: A Future Cure to Deadly Diseases? Materials Science and Engineering: C. 2017, 76: 1378-1400. Ball, R.L.; et al. Lipid Nanoparticle Formulations for Enhanced Co-delivery of siRNA and mRNA. Nano Letter. 2018, 18(6): 3814-3822. * Only for ...
Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells . FEBS Lett , 2004; 558 : 63–68.Muratovska A, Eccles MR. Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells. FEBS Lett. 2004; 558 (1–3):63–8. doi: 10.1016/S001...
GalNac-siRNA conjugate delivery technology promotes the treatment of typical chronic liver diseases Zhen-Xin Qin, Ling Zuo, Ziran Zeng, Rongguan Ma, Wenyan Xie, Xiao Zhu & show all Received 18 Nov 2024, Accepted 10 Feb 2025, Published online: 18 Feb 2025 Cite this article https:...