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GENE THERAPY IN CARDIO-VASCULAR DISEASES ; CONCEPTS, SCOPE & PERSPECTIVES

机译:心血管疾病的基因治疗;概念,范围和观点

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The genetic modification of the cells can be performed by three different methods : gene replacement, correction and augmentation. The recombinant DNA molecule of the desired gene is produced and inserted into the somatic cells either in vivo or ex vivo by various gene delivery systems namely, viral (adeno, adeno-associated or retroviruses) or non-viral (physical or chemical) methods. There are still many limitations of various delivery systems which are yet to be overcome. The different cardio-vascular diseases where gene therapy holds a promise are for the treatment of hypercholesterolemia (familial), prevention of vascular restenosis after angioplasties and for therapeutic angiogenesis (peripheral vascular angiogenesis for advanced coronary artery disease patients unfit for coronary bypass graft operation either alone or as an adjunct to CABG). The angiogenic gene therapy (AGENT) was first reported for myocardial ischemia. FGF-4 (fibroblast growth factor-4) carried by an adenoviral vector was given by intracoronary route to the patients of chronic stable angina. Gene therapy has the potential to radically affect the disease processes; however, the technique is still evolving. The main obstacle is the successful development of vectors with high transfection efficiency as well as tissue specificities.
机译:可以通过三种不同的方法对细胞进行遗传修饰:基因置换,校正和扩增。通过各种基因递送系统,即病毒(腺病毒,腺相关病毒或逆转录病毒)或非病毒(物理或化学)方法,体内或离体产生所需基因的重组DNA分子并将其插入体细胞。各种递送系统仍然存在许多限制,尚待克服。基因治疗有望实现的各种心血管疾病包括高胆固醇血症(家族性)的治疗,血管成形术后预防血管再狭窄和血管生成(晚期冠状动脉疾病患者的周围血管新生),不适合单独进行冠状动脉搭桥手术或作为CABG的附件)。血管生成基因疗法(AGENT)首次报道了心肌缺血。腺病毒载体携带的FGF-4(成纤维细胞生长因子4)通过冠状动脉内途径给予慢性稳定型心绞痛患者。基因治疗有可能从根本上影响疾病进程。但是,该技术仍在发展。主要障碍是成功开发具有高转染效率和组织特异性的载体。

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