How does gene therapy function in genetic disease treatment?

Gene therapy involves the insertion of healthy genes into cells to treat genetic diseases.

Gene therapy is a promising approach to treating genetic diseases caused by mutations in a single gene. The therapy involves the insertion of a healthy copy of the defective gene into the patient's cells to replace the faulty gene. The healthy gene can be delivered into the cells using a viral vector, which is a modified virus that can infect cells and deliver the new gene. Alternatively, the gene can be delivered using non-viral methods, such as electroporation or lipofection.

Once the healthy gene is inserted into the cells, it can produce the functional protein that was missing or defective due to the mutation. This can help to alleviate the symptoms of the genetic disease and improve the patient's quality of life. However, gene therapy is still in its early stages and there are many challenges that need to be overcome, such as ensuring that the new gene is inserted into the correct cells and that it is expressed at the right level.

Despite these challenges, gene therapy has shown promising results in clinical trials for several genetic diseases, including cystic fibrosis, haemophilia, and severe combined immunodeficiency (SCID). With further research and development, gene therapy has the potential to revolutionise the treatment of genetic diseases and offer hope to patients who previously had no effective treatment options.