Int J Med Sci 2013; 10(8):957-964. doi:10.7150/ijms.5632 This issue
1. Phase I Clinical Trial Unit, First Hospital, Jilin University, Changchun 130021, China;
2. First Hospital, Jilin University, Changchun, China;
3. Cancer Hospital of Jilin Province, Changchun, China;
4. Key Laboratory for Molecular and Engineering of Ministry of Education, Jilin University, Changchun, China.
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.
Keywords: RNA interference, hepatitis C virus, small hairpin RNA, gene therapy, HPhA