Cell-based drug screens for regulators of gene expression

Abstract From the Canadian patent

The invention provides methods and compositions for screening for pharmacological agents which regulate gene expression in mammals. An exemplary assay involves (a) contacting a mammalian cell comprising a knock-in mutant of a targeted native allele encoding a reporter of gene expression as shown in , the figure, wherein the expression of the reporter is under the control of the gene expression regulatory sequences of the native allele with a candidate , agent under conditions whereby but for the presence of the agent the reporter , is expressed at a first expression level; and (b) measuring the expression of the reporter to obtain a second expression level wherein a difference between …