Gene therapy is a technique that modifies a person’s genes to treat or cure disease. The technique can work by several mechanisms like replacing a disease-causing gene with a healthy copy of the gene or inactivating a disease causing gene that is not functioning properly. It is the introduction of genes into existing cells to prevent or cure a wide range of diseases. For example, a case of brain tumour forming by rapidly dividing cancer cells: the reason this tumour is forming is due to some defective or mutated gene. Here this technique can be applied. It can be used to modify cells inside or outside the body. When it’s done inside the body, a doctor will inject the vector carrying the gene directly into the part of the body that has defective cells. It offers the possibility of a permanent cure for any of the more than 10,000 human diseases caused by a defect in a single gene. Among these diseases, the haemophilia represents an ideal target and studies in both animals and humans have provided evidence that a permanent cure for haemophilia is within reach.

Shivangi Ojha

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