A New MicroRNA-1291 Replacement Therapy for Pancreatic Cancer Disease

DESCRIPTION (provided by applicant): Pancreatic cancer is the fourth leading cause of cancer-related death in the United States. There is a clear need for more effective therapies to treat deadly pancreatic cancer disease. MicroRNAs (miRNA or miR) are a large group of short, noncoding RNAs that control posttranscriptional gene expression in human cells, and there is increasing evidence for the existence of tumor suppressive and oncogenic miRNAs deregulated in human carcinomas. A tumor suppressor miRNA down-regulated in cancerous tissues may be reintroduced into cancer cells via a novel approach, known as miRNA replacement therapy , to block tumor growth. We have identified a tumor suppressor miRNA, miR-1291, that is expressed at reduced levels in human pancreatic ductal adenocarcinoma (PDAC) compared to normal pancreatic ductal tissue. The reintroduction of miR-1291 mimics into PDAC cells induces cancer cell cycle arrest and the suppression of xenograft pancreatic tumor growth. Given the tumor suppressi