公开(公告)号：US20100239502A1

公开(公告)日：2010-09-23

申请号：US12668551

申请日：2008-06-26

申请人： Evan Edward Santo ,  Nevenka Dimitrova ,  CHIEN Ting Chien

发明人： Evan Edward Santo ,  Nevenka Dimitrova ,  CHIEN Ting Chien

IPC分类号： A61K49/00 ,  C12N15/85 ,  C12Q1/68

CPC分类号： C12N15/1086 ,  C12Q1/6897 ,  C12Q2565/101

摘要： The invention features compositions and methods for in vivo expression analysis. The data presented herein demonstrates that ultrasound-enhanced delivery and/or expression of a composition for expression analysis comprising microbubbles vectors as well as a genetic payload, comprising a “always-on” promoter, a “reference” reporter gene, a “query” promoter and an “answer” reporter gene, enables in vivo analysis of gene expression both without requiring prior preparation (especially genetic modification) of the test subject (animal or patient) and without causing long term or systemic effects on the subject. Such an invention can be used, for example, to query the epigenotypic or phenotypic response of the individual subject to a foreign effector substance such as a pyrogen, pharmaceutical compound, pharmaceutical lead compound, an allergen, an autoimmunogene, a toxin, a polyclonal antibody, a monoclonal antibody, an antigen, a lipid, a carbohydrate, a peptide, a protein, a protein-complex, an amino acid, a fatty acid, a nucleotide, DNA, RNA, PNA, siRNA and micro RNA.

摘要翻译： 本发明的特征在于用于体内表达分析的组合物和方法。 本文提供的数据表明包括微泡载体和遗传有效载荷的用于表达分析的组合物的超声增强递送和/或表达，包括“始终在上”启动子，“参考”报告基因，“查询” 启动子和“答案”报告基因，能够在不需要测试对象（动物或患者）的事先准备（特别是遗传修饰）的情况下进行基因表达的体内分析，并且不会对受试者造成长期或全身影响。 这样的发明可以用于例如查询个体受试者对外源性效应物质如热原，药物化合物，药物引导化合物，变应原，自身免疫原，毒素，多克隆抗体的表型表型或表型反应 ，单克隆抗体，抗原，脂质，碳水化合物，肽，蛋白质，蛋白质复合物，氨基酸，脂肪酸，核苷酸，DNA，RNA，PNA，siRNA和微RNA。