The ultimate goal of cancer genetics is to exploit molecular changes in a cancer to design tumor specific therapies.Ma et al.in their recent paper in Cell Research used an elegant strategy,a CRISPR-Cas9 synthetic leth...The ultimate goal of cancer genetics is to exploit molecular changes in a cancer to design tumor specific therapies.Ma et al.in their recent paper in Cell Research used an elegant strategy,a CRISPR-Cas9 synthetic lethal knockout screen,to identify vulnerabilities in MEN1 deficient tu-mor cells in cell culture.They then successfully translated these results into the development of candidate gene targets for possible drug ther-apy.MEN1 deficiency occurs in sporadic neuroendocrine tumors,and germline mutations in the gene are the basis for cancer predisposition in the human multiple endocrine neoplasia(MEN1)syndrome 1,2.Such pa-tients are at high risk for the development of a variety of neuroendocrine neoplasms,including pancreatic neuroendocrine carcinomas,which are often not amenable for curative surgical resection and account for the mortality of MEN1 patients 3.展开更多
文摘The ultimate goal of cancer genetics is to exploit molecular changes in a cancer to design tumor specific therapies.Ma et al.in their recent paper in Cell Research used an elegant strategy,a CRISPR-Cas9 synthetic lethal knockout screen,to identify vulnerabilities in MEN1 deficient tu-mor cells in cell culture.They then successfully translated these results into the development of candidate gene targets for possible drug ther-apy.MEN1 deficiency occurs in sporadic neuroendocrine tumors,and germline mutations in the gene are the basis for cancer predisposition in the human multiple endocrine neoplasia(MEN1)syndrome 1,2.Such pa-tients are at high risk for the development of a variety of neuroendocrine neoplasms,including pancreatic neuroendocrine carcinomas,which are often not amenable for curative surgical resection and account for the mortality of MEN1 patients 3.