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What can be Treated with Gene Therapy?

Gene Therapy

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While gene therapy may seem like a new innovation in the field of medical science, you will be surprised to know that it was first performed in 1980 by Martin Cline. Further, the first successful nuclear gene transfer in humans was performed in May 1989. Between 1989 and February 2016, over 2,300 clinical gene therapy trials have been conducted.

Gene therapy holds the potential to transform medicine and find the cure for several chronic diseases, even cancer.

What is Gene Therapy?

Gene Therapy is a medical technique that uses genes to treat or prevent diseases. This technique is performed by introduction of genetic material into cells to compensate for abnormal genes. For instance, when a mutated gene leads to production of a faulty protein or doesn’t synthesize the protein at all, gene therapy may help to introduce a normal copy of the gene, to restore the function of the protein.

In simple terms, gene therapy is when DNA (genetic material) is introduced into a patient to treat a genetic disease. This new DNA usually contains a functioning gene which corrects the effects of a faulty or disease-causing gene mutation.

Gene Therapy for Diseases

Gene therapy treatment has witnessed fast paced developments from the conceptual stage to laboratory research to clinical trials to treat chronic diseases.Here are several diseases for which gene therapy has been found effective: –

Severe Combined Immune Deficiency (SCID)

The children who suffer from SCID are born without an effective immune system. When the immune system doesn’t function properly, the body becomes prone to infections, and it becomes difficult to battle infections. Research has found gene therapy to be effective in treating this disorder. A therapeutic gene called ADA is introduced into the bone marrow cells, undergoing this, patients have been known to lead a normal life without any noticeable side effects.

Chronic Granulomatus Disorder (CGD)

Chronic granulomatous disease (CGD) is a genetic disorder in which certain immune system cells get mutated and do not function properly. This disorder leads to repeated and severe infection, and can be fatal. Using similar gene therapy as in SCID, this disorder can be effectively treated.


Haemophilia is an inherited genetic disorder that leads to incapability to form blood clots and stop bleeding. Due to this disorder, patients can suffer from external and internal bleeding, which can be life threatening. Clinical trials have been conducted in which a therapeutic gene was introduced into the patient’s liver, which leads to normal blood clotting.


Cancer involves abnormal cell growth which can spread to other parts of the body. Numerous gene therapy approaches have been developed to treat a wide variety of cancers. The therapy is still under various clinical trials, and uses a patient’s own immune cells, called T-cells, along with white blood cells.

Neurodegenerative Diseases

Neurodegeneration occurs when there is progressive loss of structure or function of neurons, and can even lead to death of neurons. Neurodegenerative processes lead to many diseases including Parkinson’s, Alzheimer’s, Huntington’s, etc. Recent gene therapy trials have proved to be effective in treating neurodegenerative disorders.

Gene therapy techniques have been applied to treat a range of genetic and acquired disorders such as viral infections, heart disease, diabetes, and more. Some of these have proved effective in clinical trials, while some are still in the early phase of clinical trials.

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