What is the purpose of gene therapy?

The purpose of gene therapy is to treat or prevent diseases by altering or replacing faulty genes in the body.

Gene therapy is a revolutionary medical technique that aims to treat or prevent diseases by modifying the genetic material within an individual's cells. The concept is based on the understanding that many diseases, including certain types of cancer, cystic fibrosis, and some inherited disorders, are caused by problems in the DNA. These problems can be due to mutations, deletions, or other types of genetic errors that result in the production of faulty proteins or no proteins at all.

The process of gene therapy involves introducing, removing, or altering genetic material within a person's cells. This is typically achieved by using a vector, usually a virus that has been genetically engineered to carry healthy copies of the gene into the cells. Once inside the cells, the new gene can start producing the correct protein, thereby correcting the genetic disorder or disease.

There are two main types of gene therapy: somatic and germline. Somatic gene therapy involves altering the genes in body cells that do not contribute to the production of the next generation, meaning the changes are not heritable. This is the most common type of gene therapy and is currently the only type used in humans. Germline gene therapy, on the other hand, involves changing genes in eggs or sperm, or in the cells that produce them. The changes made in this type of therapy would be passed on to future generations.

Gene therapy holds great promise for the treatment of a wide range of diseases. However, it is still a relatively new field and there are many technical and ethical challenges to overcome. For example, delivering the gene to the right cells without causing damage or an immune response is a major challenge. Additionally, there are concerns about the potential for unintended consequences, such as the development of cancer if the new gene inserts itself in the wrong place in the genome. Despite these challenges, the potential benefits of gene therapy make it a highly active area of research.