What is the role of gene therapy in treating diseases?

Gene therapy is used to treat diseases by replacing, inactivating or introducing genes into cells to correct genetic disorders.

Gene therapy is a relatively new and rapidly evolving field of medicine that holds great promise for treating a variety of diseases. The basic idea behind gene therapy is to use DNA as a pharmaceutical agent to treat disease. It works by introducing, replacing, or inactivating specific genes to help the body fight or prevent disease.

There are two types of gene therapy: somatic and germline. Somatic gene therapy involves altering the genes in body cells, such as bone marrow or blood cells. This type of therapy can treat the individual patient, but the changes are not passed on to the next generation. On the other hand, germline gene therapy involves changing genes in eggs or sperm, or in the cells that produce them. These changes are inherited by future generations.

Gene therapy can be used to treat a variety of diseases, including inherited disorders, some types of cancer, and certain viral infections. For example, it's being used to treat diseases like cystic fibrosis, haemophilia, and even some cancers. In these cases, the faulty gene is replaced with a healthy one, allowing the body to function normally.

The process of gene therapy involves creating a piece of DNA with the correct, healthy version of the faulty gene. This is then inserted into a vector, usually a virus that has been genetically modified to carry human DNA. The vector is used to deliver the DNA into the patient's cells. If successful, the new gene will make a functional protein to correct the problem, effectively curing the disease.

However, gene therapy is still in its experimental stages and is currently used only to treat diseases for which no other treatments are available. It's a complex process with many potential risks and complications, including immune reactions, inflammation, and even the possibility of causing cancer. Despite these challenges, the potential benefits of gene therapy make it a promising field for future medical treatments.