Induction of p53 and up-regulation of the p53 pathway in the human 5q- syndrome.

We have demonstrated induction of p53 and up-regulation of the p53 pathway in the human 5q– syndrome. This most probably results from haploinsufficiency of the ribosomal gene RPS14. The ribosome biogenesis checkpoint that results in activation of p53 protein and up-regulation of the p53 pathway offers a new potential therapeutic target in the human 5q– syndrome. However, this will be an option only if this intervention does not abrogate the critical tumor suppressor function of p53