The transfer and expression of human clotting factor IX in muscle mediated by electroporation

Objective To probe into the feasibility of increasing hFⅨ cDNA transfer and expression in muscle. Methods The high-frequency electric field was used to promote both Lac-Z-encoding plasmid pCMVβ and hFⅨ-expressing plasmid G1NaMCⅨ to transfer and express in muscle. The effects of frequency and length of square pulse,as well as eletroporation time on hFⅨ expression were investigated. Results Electric stimulation could increase the transfer and expression of pCMVβ in muscle,the number of X-gal positive myofiber cells in electroporation-treated mice is 2.1 times larger than that of mice not treated by electroporation (P0.01).The most optimal electric simulation condition for hFⅨ cDNA transfer and expression was obtained, under this condition, the highest level of hFⅨ antigen in plasma is (40±5.4) ng/ml and 7 times higher than that of mice without electroporation(P0.001). Conclusion Electroporation is able to enhance hFⅨ cDNA transfer and expression in muscle efficiently.