SARS基因治疗的核苷酸干扰(siRNA)策略  

Strategy for siRNA based gene therapy of SARS

作　　者：孟夏[1] 胡放 邱祺宏[1] 叶迅[1] 梁旻 陈红专[1] 

机构地区：[1]上海第二医科大学药理学教研室,上海200025 [2]上海三维生物技术有限公司,上海201206

出　　处：《中国药理学通报》2003年第7期723-726,共4页Chinese Pharmacological Bulletin

摘　　要：新型冠状病毒是严重急性呼吸道综合征 (SARS)的病原体 ,目前缺乏有效预防和治疗药物。作者在研究SARS冠状病毒基因的基础上 ,借鉴近年迅速发展的siRNA (smallinterferingRNR)基因药物的思路 ,经过对制剂和给药途径的分析 ,提出针对SARS有效、安全、特异的siRNA药物设计策略。通过PEI -siRNA质粒复合物的喷雾给药 ,将该药物靶向性地导入肺部 ,进入肺部细胞并表达特异性的siRNA ,对已经感染病毒的细胞内部的病毒基因进行沉默 ,抑制病毒的进一步复制和繁殖 ,既达到治疗目的 ,且克服siRNA药物中靶向性和导入性差的两大困难 ,提供了一条安全。A newly discovered member of the coronavirus family has been considered to be the cause of severe acute respiratory syndrome (SARS) There are no effective drugs for cure and prevention of this syndrome at this moment Based on the known genomic sequences of coronavirus, the authors oropose here a plasmid vector expressed siRNA strategy for SARS gene therapy Aerosol delivery of plasmid DNA to the lungs offers the possibility for direct application of gene preparations to pulmonary surfaces, especially in conjunction with polyethyleneimine (PEI, a polycationic polymer). The complexes of PEI DNA may result in a specific high level of pulmonary transfection and resolve the two key difficulties of SiRNA both in delivering and targeting This strategy might show great potential for SARS gene therapy

关 键 词：SARS 冠状病毒 SIRNA PEI 喷雾疗法 

分 类 号：Q7[生物学—分子生物学] Q81