OBJECTIVE: To accomplish a kind of therapeutic gene for @entity447 , and observe the expression of @entity1 factor @entity797 ( @entity2801 ) in vivo. METHODS: @entity1 clotting factor @entity797 cDNA with B-domain deleted (Delta760aa approximately 1639aa) was inserted into vector pRC/ @entity5842 to form pRC/ @entity5842 - @entity2801 BD, which conjugated with in vivo liposome transfection reagent ( @entity11505 - @entity165 ) to accomplish a kind of therapeutic gene, pRC/ @entity5842 - @entity2801 BD- @entity11505 - @entity165 . @entity19 were injected with pRC/ @entity5842 - @entity2801 BD- @entity11505 - @entity165 i.m. and sacrificed 48 hours, 10 days, 20 days, 30 days, 40 days and 50 days later, respectively. Tissues such as heart, liver, spleen, lung, kidney and muscle were harvested, the distribution and transcription as well as expression of @entity2801 BD cDNA were detected by means of PCR, RT-PCR and immunohistochemistry techniques. In addition, the antigen and antibody of @entity2801 in plasma were measured. RESULTS: There was high expression of @entity2801 in plasma and tissues at the 48(th) hour after injection. On day 10, antigen level of @entity2801 in plasma reached its peak, 17.55 ng/ml, and gradually reduced later. The antibody of @entity2801 in plasma emerged on day 10 after injection, and increased and gradually reached 37.06 U/ml on day 50 after injection. PCR, RT-PCR and immunohistochemistry showed that @entity2801 BD cDNA and its transcription as well as expression existed in all kinds of tissues, and lasted longer in spleen, lungs and kidneys than in heart, liver and muscle. CONCLUSION: Therapeutic gene, pRC/ @entity5842 - @entity2801 BD- @entity11505 - @entity165 , produced by combination of pRC/ @entity5842 - @entity2801 BD and @entity11505 - @entity165 liposome can express @entity1 F @entity797 successfully in vivo, which lays an experimental foundation for curing @entity447 by gene-drug in clinic.

[Preliminary experimental research on gene therapy for XXXX ].

@entity1 :: ('9606', 'Species') :: ['human', 'Human'] @entity2801 :: ('2157', 'Gene') :: ['hF VIII'] @entity5842 :: ('11886', 'Species') :: ['RSV'] @entity11505 :: ('MESH:C070046', 'Chemical') :: ['DOTAP'] @entity447 :: ('MESH:D006467', 'Disease') :: ['hemophilia A'] @entity19 :: ('10090', 'Species') :: ['Mice'] @entity797 :: ('1351', 'Gene') :: ['VIII'] @entity165 :: ('MESH:D002784', 'Chemical') :: ['Cholesterol']
@entity447 :: (MESH:D006467,Disease) :: ['hemophilia A']
