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*Canine and murine models are dogs and mice respectively. They are models with hemophilia that closely mimic the human disease and are useful for study and treatment of this disease.
*Factor IX and Factor VII are two of the coagulation factors, (proteins). These factors are part of a cascade of events, in the bloodstream, that are necessary for blood clot formation.
Experiment 1:
An intron is a non-coding sequence present in most eukaryotic genes. After transcription, the intron is spliced out of the mRNA as a post-transcriptional modification, before it is considered a mature mRNA.
LTRs are are located at the ends of retrovirus and are short sequence that contain the transcription start signals and other regulatory sequences, They also contain the signals for the 3' cleavage and polyadenylation of the mRNA.
Xba I is a restriction endonuclease, (r.e.), that recognizes the sequence T/CTAGA. The r.e. cleaves where the / is in this sequence.
Intra-portal infusion means that the site of infusion is in a vein leading to the liver.
Autoradiography is an x-ray of the gel. The bound radioactive nucleic acid sequences will show as a dark band on the x-ray.
A Southern Blot Analysis is an assay that detects the presence of DNA. The assay is performed by hybridizing a complementary radiolabeled probe to the DNA strand of interest. The results are viewed through autoradiography.
ELISA stands for Enzyme-linked immunoabsorbent assay. This is an enzymatic test and the purpose is to detect an antibody. It is a sandwich technique where the antibody to be detected binds to an antigen, and then a second, enzyme-labeled antibody directed against the antibody to be detected is added. The substrate of the enzyme is added and a colored reaction product is formed, which can then be read spectrophotometrically. In the case of this study, the antibody to be detected would be against hFIX.
PTT stands for Partial Thromboplastin time. This test measures the intrinsic pathway of the coagulation cascade. Factor IX is an important part of this cascade. In vitro this test works by measuring the time needed for a fibrin clot to form following the addition of calcium and partial thromboplastin.