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Gene therapy targeting p53

机译:靶向p53的基因疗法

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The product of the p53 gene plays a critical role in the regulation of cell growth. Mutations of this gene are associated with transformation to a malignant phenotype. Correction of the gene defect through transfer of a wildtype p53 gene into malignant cells, or targeting malignant cells with oncolytic viruses (ONYX-015) genetically engineered to proliferate in cells containing mutant genes has been identified as a therapeutic approach by preclinical assessment. Initial clinical trials have confirmed functional activity and expression of the transgene product in Adp53-injected malignant tissue and tumor specific viral proliferation have been observed in patients receiving intratumoral injection of ONYX-015.
机译:p53基因的产物在调节细胞生长中起关键作用。该基因的突变与转化为恶性表型有关。通过临床前评估,通过将野生型p53基因转移到恶性细胞或用经基因工程改造以在含有突变基因的细胞中增殖的溶瘤病毒(ONYX-015)靶向恶性细胞来纠正基因缺陷已被确认为治疗方法。最初的临床试验已经证实,在接受瘤内注射ONYX-015的患者中观察到功能活性和转基因产物在Adp53注射的恶性组织中的表达,并且观察到肿瘤特异性病毒增殖。

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