Chordoma Foundation

Evaluation of miRNA-1 as a potential therapeutic target for treatment of chordoma

Dr. DuanZhenfeng Duan, MD, PhD
Sarcoma Molecular Biology Laboratory
Massachusetts General Hospital$25,000 seed grant

MicroRNAs (miRNAs) are important regulators of gene and protein expression, and abnormal expression of these molecules may play a role in the development of human cancer. Dr. Duan’s group has previously found that miRNA-1 in particular is expressed at abnormally low levels in chordoma tissues compared with in healthy tissues, and the goal of this project is to understand the clinical consequences of this low miRNA-1 expression. Researchers will first assess levels of miRNA-1 expression in chordomas and correlate them with clinical outcomes. Because a single miRNA may regulate a number of genes and proteins, researchers will catalogue all the genes in chordoma cells that are regulated by miRNA-1 in order to better understand the effects of miRNA-1 deficiency. Finally, the researchers will administer miRNA-1 to mice grafted with chordoma tumors to test whether restoring miRNA-1 expression to normal levels could have an anti-tumor effect. Ultimately, these experiments could help to identify a novel, targeted treatment approach for chordoma and other cancers.

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