重组腺相关病毒2人凝血因子IX无创途径给药治疗血友病B的研究.pdf

本研究证实灌肠途径灌注rAAV2／hFIx后血友病B小鼠体内可表 达具有凝血活性的hFIX，表明结肠灌注无创给药可能成为血友病B 基因治疗的新途径。 关键词2型重组腺相关病毒，凝血因子Ix，血友病B小鼠，基因 治疗，灌肠 H ABSTRACT BisanX—linked caused Hemophilia disease bya hemorrhagic offunctional factor isthe deficiency coagulant therapy IX(FIX)，gene idealmethodtocure routesof FIXfor it．However,the present delivering B all toestablish are invasive．Inorder a hemophiliagenetherapy noninvasive administration，wedesigned therapyby colon-targetedgene ourrecentstudieshaveindicatedthatcolonic coloclysis，and epithelial transfected cells rAAV2／hFIXcan hFIXwith by express coagulant in activitiesvitro．Inthis ordertofurther whether study,in investigate hFIXwitll activitiescallbe in enemawith coagulant vivo expressedpost were at ∥LW2mFIX．normalmice rA√W2／llFIX Kunming firstlygiven x 1 l thedoseof andthe levelsofhFIX O”vg／kgbycoloclysisexpression were thebasisof B were analyzed．On mice given