Gene Therapy for Diseases


Over the past two decades, there has been a tremendous advancement in Gene Therapy. Since the first attempt of modifying human DNA in 1980 till now, several trials have been conducted while many are still underway to ensure that any gene therapy that is brought into the clinic is safe as well as effective. Some of the most successful advancements of Gene therapy for diseases are:

  1. Severe Combined Immune Deficiency (SCID):

This deadly genetic disorder known the SCID was one of the first genetic disorders to be treated successfully with gene therapy, proving that the approach could work.

  1. Chronic Granulomatus Disorder (CGD):

CGD is a genetic disease that affects the immunity of the patients. It is basically the disease in the immune system that takes away the patient’s ability to fight against microbial infections that can be dangerous. The Gene Therapy enabled the reconstitution of the immune systems providing protection against bacterial and fungal infections.

  1. Hereditary blindness:

Gene therapies are being developed to treat varied types of inherited blindness, especially the degenerative forms. The years long research shows that gene therapy has the potential to slow or even reverse vision loss.

  1. Haemophilia:

Haemophilia is a disease where the blood does not clot and the patent suffer from continuous bleeding that can be fatal. This happens because the protein that helps the blood form clots is missing. In a small trial for the cure by gene therapy,the researchers successfully delivered a gene for Factor IX, the missing clotting protein, to liver cells.

  1. Cancer:

There is a wide variety of cancers and thus, multiple gene therapy strategies have been evolved to cure them. Among the thousands of gene therapy trials, 2/3rds have been made to treat cancer and several of these are already in their advanced stages.

  1. Neurodegenerative Diseases:

There has been the recent advancement in Gene Therapy for the treatment of brain related diseases such as Parkinson’s and the Huntington’s disease. This is basically the loss of the cells in brain that produce the signalling molecule dopamine and the patient loses the ability to control movements. The Gene therapy allows introducing such genes into cells in a small area of brain and provides improved muscle control to the patients.


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