Gene therapy is a medical technique that involves changing or fixing genes inside a person’s cells to treat or prevent diseases. It works by adding, removing, or altering genetic material to correct faulty genes that cause illness.
Here’s how it works simply:
- Problem in genes:
Some diseases happen because a gene is missing or not working properly. For example, genetic disorders like cystic fibrosis or muscular dystrophy occur due to defective genes. - Goal of gene therapy:
The aim is to fix the problem at its source — the gene — instead of just treating the symptoms. - Methods used:
- Replacing a faulty gene: A normal copy of the gene is added to take over the job of the damaged one.
- Inactivating a harmful gene: A gene that causes disease is turned off.
- Introducing a new gene: A completely new gene is added to help the body fight disease (for example, to make immune cells better at attacking cancer cells).
- How it’s delivered:
Scientists usually use harmless viruses or other delivery systems (called vectors) to carry the healthy gene into the patient’s cells. - Applications in medicine:
- Treating inherited diseases like hemophilia and sickle cell anemia
- Helping immune cells target cancer (CAR-T cell therapy)
- Treating some types of blindness and immune system disorders
In simple words, gene therapy fixes diseases by repairing or replacing the broken instructions inside our cells, offering a way to treat illnesses that were once thought to be untreatable.