Gene editing to treat tumours mediated by fusion genes

Sandra Rodríguez

CNIO, Centro Nacional de Investigaciones Oncológicas, Spain

Cancer is currently one of the primary medical problems in the developed world. There are more than 120 types of cancer, and the different types of cancer are treated using a combination of chemo, radiation therapy, surgery, and, more recently, targeted therapies and immunotherapy, but these treatments have variable results and can cause serious side effects. The absence of effective therapies and the acquired resistance to existing treatments makes the development of effective treatment for certain types of cancer one of the main goals of innovative therapies.

About 20 % of cancers, including colon, prostate and leukemia cancers, are characterized by the presence of hybrid genes known as fusion genes. Fusion genes represent specific molecules of the tumor cell and are therefore promising therapeutic targets in the battle against cancer. This project uses a therapeutic approach based on gene editing for the treatment of tumors induced by fusion genes. The results obtained so far show a reduction in the size of tumors, in animal models, of sarcoma and leukemia.

Considered one of the great advances in biotechnology, gene editing is a powerful tool that could radically change cancer treatment. This project opens the way for other antineoplastic treatments based on this technology.