GENE THERAPY USING INTRA-HEPATIC METHODGENE THERAPY INTRA-HEPATIC METHOD

Texas Biomedical Research Institute, San Antonio TX

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Abstract

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Hemophilia is a inherited condition in humans characterized by the absence of factor IX which is required to complete the process of clot formation to prevent blood loss during trauma and surgery. Since this condition is caused by a deffective sex-linked ressesive gene, there is a strong possibility of correcting the deffect by gene therapy. Therefore, the objective of this study is to determine the technical feasibility of retrograde venous delivery of plasmids containing human gene that codes for factor IX to the liver. Liver is the major source of all clotting factors and therefore is the target organ for this procedure. In addition, to the plasmid encoding for factor IX , a second plasmid encoding for an enzyme called integrase will be administered at the same time. Integrase will aid in inserting the factor IX gene in to the host cell DNA. Successful integration of the factor IX gene and production of human factor IX by the baboon liver will be determined by blood tests. If this strategy appears safe and functional, then it can be used for the treatment of factor IX deficient hemophilia in humans.