Human Health - Gene Therapy

The technique of treating genetic disorders by correcting defective genes in somatic cells is called gene therapy.

• Gene therapy is the treatment of diseases by replacing, altering, or adding genes to correct genetic defects.
• It is used to treat genetic disorders such as haemophilia, cystic fibrosis, sickle cell anaemia, and thalassemia.
• Genes can be delivered into cells by ex vivo (outside the body), in vivo (inside the body), or using vectors like viruses and liposomes.
• There are two types: germ-line therapy (affects future generations, not used in humans) and somatic cell therapy (affects only the patient and is commonly used).
• Gene therapy has applications in treating genetic diseases, cancer, and improving immune response, but it involves technical and ethical challenges.