发明名称 Efficient methods for assessing and validating ecandidate protein-based therapeutic molecules encoded by nucleic acid sequences of interest
摘要 A method of determining at least one quantitative or qualitative pharmacological, physiological and/or therapeutic, parameter or effect of a recombinant gene product in vivo, the method comprises (a) obtaining at least one micro-organ explant from a donor subject, the micro-organ explant comprising a population of cells, the micro-organ explant maintaining a microarchitecture of an organ from which it is derived and at the same time having dimensions selected so as to allow diffusion of adequate nutrients and gases to cells in the micro-organ explant and diffusion of cellular waste out of the micro-organ explant so as to minimize cellular toxicity and concomitant death due to insufficient nutrition and accumulation of the waste in the micro-organ explant, at least some cells of the population of cells of the micro-organ explant expressing and secreting at least one recombinant gene product; (b) implanting the at least one micro-organ explant in a recipient subject; and (c) determining the at least one quantitative or qualitative pharmacological, physiological and/or therapeutic, parameter or effect of the recombinant gene product in the recipient subject.
申请公布号 US2003124565(A1) 申请公布日期 2003.07.03
申请号 US20020190754 申请日期 2002.07.09
申请人 GARFINKEL LEONARD I.;PEARLMAN ANDREW L.;MITRANI EDUARDO N. 发明人 GARFINKEL LEONARD I.;PEARLMAN ANDREW L.;MITRANI EDUARDO N.
分类号 C12N15/10;(IPC1-7):C12Q1/68;A61K49/00 主分类号 C12N15/10
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