通过组蛋白基因载体 HPhA 递送的小发夹 RNA 抑制 HCV 5'-NTR 和核心表达。
Inhibition of HCV 5'-NTR and core expression by a small hairpin RNA delivered by a histone gene carrier, HPhA.
机构信息
Phase I Clinical Trial Unit, First Hospital, Jilin University, Changchun 130021, China.
出版信息
Int J Med Sci. 2013 Jun 9;10(8):957-64. doi: 10.7150/ijms.5632. Print 2013.
siRNA (small interfering RNA) interference represents an exciting new technology that could have therapeutic applications for the treatment of viral infections. However, a major challenge in the use of siRNA as a therapeutic agent is the development of a suitable delivery system. We demonstrated that a new non-viral transgene carrier, recombinant archaeal histone from the hyperthermophile Pyrococcus horikoshii OT3 (HPhA), can transfect short hairpin RNA (shRNA) expressing plasmids into HL-7702 cells to inhibit the expression of HCV 5'NTR and Core protein and mRNA. Plasmids Psilencirle transfected by HPhA inhibited the expression of HCV 5'-NTR and Core protein and mRNA in HL-7702 cells. The transfection efficiency of HPhA in HL-7702 cells was not affected by 10% fetal calf serum (FCS). HPhA exhibited effects of transfection without apparent toxicity, and with high affinity for DNA. This suggests that HPhA may be useful for RNAi-based gene therapy in vivo.
siRNA（小干扰 RNA）干扰代表了一种令人兴奋的新技术，可能在治疗病毒感染方面具有治疗应用。然而，将 siRNA 用作治疗剂的主要挑战是开发合适的传递系统。我们证明了一种新的非病毒转基因载体，来自高温古菌 Pyrococcus horikoshii OT3（HPhA）的重组古菌组蛋白，可以将短发夹 RNA（shRNA）表达质粒转染到 HL-7702 细胞中，以抑制 HCV 5'NTR 和核心蛋白和 mRNA 的表达。由 HPhA 转染的 Plasmids Psilencirle 抑制了 HL-7702 细胞中 HCV 5'-NTR 和 Core 蛋白和 mRNA 的表达。HPhA 在 HL-7702 细胞中的转染效率不受 10%胎牛血清（FCS）的影响。HPhA 表现出转染作用而没有明显的毒性，并且与 DNA 具有高亲和力。这表明 HPhA 可能对体内基于 RNAi 的基因治疗有用。
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