Gene Therapy

By Sanam Marjan

In the world of technological advances, it has become possible to rectify a person’s genetic makeup in order to avoid diseases and underlying genetic problems. Gene therapy allows people to recover without taking drugs or by undergoing surgery and is often called gene transfer or gene addition.

It was developed in order to:

• Introduce a gene into cells to help fight a particular disease.

• Introduce a non-faulty copy of a gene to stand in for the altered copy causing the disease.

Gene contains our DNA which is the code that controls much of our body’s form and function. From making us tall to the regulation of our body systems, genes code for all. And Genes that don’t work properly and have slightly dysregulated code may cause diseases. So, gene therapy is used to replace the faulty gene or adds a new gene in an attempt to improve or cure our body’s ability to fight disease.

Why is it being done?

• To replace mutated genes: Due to genes working incorrectly, certain cells become diseased, which can be corrected by the replacement of this defective gene to help treat the disease.

• To fix mutated genes: Mutated genes that can cause diseases could be turned off. And healthy genes that help prevent disease could be triggered in order to inhibit the disease.

• To make diseased cells more evident to the immune system: In some cases, our immune system doesn’t attack diseased cells because it fails to recognise them as intruders. Hence gene therapy can be used to train our immune system to recognise the cells that are a threat.

Cystic fibrosis, AIDS, haemophilia and other inherited diseases which can be passed from parents to offsprings can be treated using gene therapy. Moreover critical life threatening diseases such as diabetes, heart disease and cancer can be also treated.

Gene therapy is currently a part of a clinical trial in the United States, but many countries have now started to offer the service at a premium price.