Tokcan Berkay, Demirtaş Esra Nur, Sözer Selçuk
Department of Genetics, Aziz Sancar Institute of Experimental Medicine, Istanbul University, Gureba Str., Capa Campus, No:69, 34093, Sehremini/Istanbul, Türkiye.
Institute of Health Sciences, Istanbul University, 34093, Istanbul, Türkiye.
Immunogenetics. 2025 Feb 28;77(1):16. doi: 10.1007/s00251-025-01374-y.
Philadelphia chromosome-negative myeloproliferative neoplasms (Ph-MPNs) are characterized by the overproduction of myeloid cells and a lack of response to cytokine signaling, along with genomic instability and the accumulation of nucleic acids in the cytoplasm. In this study, we investigated the effects of oligonucleotide-gold nanoparticle conjugates (ON-GNPs) targeting JAK2 or JAK2V617F mRNAs on nucleic acid-sensing pathways in HEL, SET2, and K562 cell lines. We evaluated changes in gene expression related to TLR9 and cGAS/STING pathways, RAGE/TLR9 receptor dynamics, and inflammatory cytokine release over short-term (0.5-2 h) and long-term (24-72 h) exposures. Our results demonstrated that ON-GNPs transiently suppressed TLR9, IRF7, and NFKB1 expression during the short term, followed by significant upregulation after 24 h, persisting up to 72 h. Notably, JAK2V617F-targeting ON-GNPs induced heightened IRF7 activation in HEL and SET2 cells after 24 h without affecting TLR9/RAGE expression. Additionally, IL-8 secretion increased in HEL and SET2 culture media after 72 h, correlating with interferon pathway activation. This study reveals that complementary ON-GNPs can modulate nucleic acid-sensing pathways, suppressing IL-8 and inflammatory signaling in the short term while inducing delayed activation of TLR9 and IRF7 in the presence of JAK2V617F. These findings provide a promising foundation for developing ON-GNP-based therapeutic strategies to manage inflammation and disease progression in Ph-MPNs.
费城染色体阴性骨髓增殖性肿瘤(Ph-MPNs)的特征是髓系细胞过度产生、对细胞因子信号缺乏反应,同时伴有基因组不稳定和细胞质中核酸积累。在本研究中,我们研究了靶向JAK2或JAK2V617F mRNA的寡核苷酸-金纳米颗粒偶联物(ON-GNPs)对HEL、SET2和K562细胞系中核酸感应途径的影响。我们评估了短期(0.5-2小时)和长期(24-72小时)暴露后与TLR9和cGAS/STING途径、RAGE/TLR9受体动力学以及炎性细胞因子释放相关的基因表达变化。我们的结果表明,ON-GNPs在短期内短暂抑制TLR9、IRF7和NFKB1的表达,随后在24小时后显著上调,并持续至72小时。值得注意的是,靶向JAK2V617F的ON-GNPs在24小时后诱导HEL和SET2细胞中IRF7激活增强,而不影响TLR9/RAGE表达。此外,72小时后HEL和SET2培养基中IL-8分泌增加,与干扰素途径激活相关。本研究表明,互补的ON-GNPs可以调节核酸感应途径,在短期内抑制IL-8和炎性信号,同时在存在JAK2V617F的情况下诱导TLR9和IRF7的延迟激活。这些发现为开发基于ON-GNP的治疗策略以控制Ph-MPNs中的炎症和疾病进展提供了有希望的基础。