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Gene Therapy in Cancer Treatment


Gene therapy is a still somewhat experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, haemophilia, Parkinson’s disease, cancer and HIV, via a number of different approaches.

A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy, but it has been used with some success.

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.

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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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Pioneering Genetic Therapy Cures One-Year-Old of Acute Lymphoblastic Leukaemia (ALL)


Layla-RichardsA one-year-old girl, tiny Layla Richards, has become the first person in the world to receive a “designer” immune cell therapy to cure her aggressive “incurable” cancer.

Layla was a mere 14-weeks-old when her mother, Lisa Foley, noticed that Layla’s heart was beating rather fast and that she was constantly crying and hardly eating. Doctors initially suspected that Layla might have a stomach bug, but a blood test diagnosed an aggressive case of a common childhood cancer, Acute Lymphoblastic Leukaemia (ALL).

Layla was immediately to an intensive care unit at Great Ormond Street Hospital (GOSH) in London and treatment was started immediately, but as is often the case with such an aggressive cancer, neither the chemotherapy nor the bone marrow transplant that followed worked. The problem in such aggressive cancers is that cancer cells can remain hidden or resistant to drug therapy. Read the rest of this entry

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