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Gene Therapy: Does it Work?


While Gene Therapy has been around for a few years already, we don’t seem to be hearing much about it being used to treat cancer, especially paediatric cancer, and one cannot help but wonder why…

In most gene therapy studies, a “normal” gene is inserted into the genome to replace an “abnormal,” disease-causing gene. In cancer, some cells become diseased because certain genes have been permanently turned off. Using gene therapy, mutated genes that cause disease could be turned off so that they no longer promote disease, or healthy genes that help prevent disease could be turned on so that they can inhibit the disease.

Other cells may be missing certain genes. Researchers hope that replacing missing or defective genes can help treat certain diseases. For example, a common tumor suppressor gene called p53 normally prevents tumor growth in your body. Several types of cancer have been linked to a missing or inactive p53 gene. If doctors could replace p53 where it’s missing, that might trigger the cancer cells to die.

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