GENE BASED THERAPEUTIC AGENT FOR PULMONARY HYPERTENSION

描述：(从应用程序修改)。基因疗法可能会取代 慢性持续静脉留置导管和持续输液，或 另一种肺移植，用于治疗原本致命的 原发性肺动脉高压病。现建议： 将前列腺素合成酶(PGHS)基因导入肺部将 通过增加肺血管反应性而增加 前列环素和前列腺素E_2。肺血管内皮细胞的转染法 通过静脉注射转基因作为阳离子脂质粒 这些调查人员此前已经证明了这一复杂性。第一阶段 将决定肺血管的反应性 可通过体内PGHS基因转移通过静脉或 或正常动物的呼吸道内路线。第二阶段的研究将是 在肺动脉高压的动物模型中进行。初级阶段的研究 肺动脉高压被认为是初步的安全和有效的 针对更大范围的继发性肺病患者的研究 高血压和肺心病。 建议的商业应用：不可用

DESCRIPTION: (Modified from application). Gene therapy may replace chronic constant intravenous catheter and continuous infusion, or the alternative lung transplantation, for the treatment of the otherwise fatal disease of primary pulmonary hypertension. It is proposed that transfection of the lungs with the prostaglandin synthase (PGHS) gene will decrease pulmonary vascular reactivity by increasing generation of prostacyclin and PGE2. Transfection of the pulmonary vascular endothelium by intravenous administration of a transgene as a plasmid cationic lipid complex has previously been demonstrated by these investigators. Phase I of the proposed study will determine whether pulmonary vascular reactivity can be decreased by in vivo PGHS gene transfer through either intravenous or intra-airway routes in normal animals. Phase II studies will be conducted in animal models of pulmonary hypertension. Studies of primary pulmonary hypertension are considered preliminary safety and efficacy studies for the much larger group of patients with secondary pulmonary hypertension and cor pulmonae. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE