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

使用肝内方法的基因治疗 肝内方法基因治疗

• 批准号: 8172694
• 负责人: Krishna K Murthy
• 金额: $ 14.82万
• 依托单位: TEXAS BIOMEDICAL RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-05-01 至 2011-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8172694
• 关键词: Blood Coagulation Factor; Blood Tests; Cells; Coagulation Process; Code; Computer Retrieval of Information on Scientific Projects Database; DNA; Enzymes; Factor IX; Funding; Genes; Grant; Hemophilia A; Hemorrhage; Hepatic; Human; Inherited; Institution; Integrase; Link; Liver; Methods; Operative Surgical Procedures; Organ; Papio; Plasmids; Procedures; Process; Production; Research; Research Personnel; Resources; Source; Time; Trauma; Treatment Factor; United States National Institutes of Health; Venous; gene therapy; prevent; sex

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.

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 血友病是人类的一种遗传性疾病，其特征是缺乏凝血因子IX，该因子是完成凝块形成过程以防止创伤和手术期间失血所必需的。由于这种情况是由一种与性别相关的缺陷基因引起的，因此有很大的可能性通过基因治疗来纠正这种缺陷。因此，本研究的目的是确定逆行静脉注射含有编码人凝血因子IX基因的质粒到肝脏的技术可行性。肝脏是所有凝血因子的主要来源，因此是这一过程的靶器官。此外，对于编码因子IX的质粒，将同时注射编码一种称为整合酶的酶的第二个质粒。整合酶有助于将因子IX基因插入到宿主细胞DNA中。因子IX基因的成功整合和人类因子IX的产生将通过血液测试来确定。如果这种策略看起来安全有效，那么它可以用于治疗人类凝血因子IX缺乏性血友病。