Gene therapy encompasses a variety of techniques that attempt to fix genetic defects in a person鈥檚 cells. These techniques usually involve using viruses to insert healthy copies of genes into the cells of people who carry one or more faulty copies of an important gene.
This branch of medicine initially hit serious setbacks, including cancer and harmful immune responses in some patients. Decades later, researchers now use safer, more sophisticatedly engineered viruses for gene transfer.
So far, only a few gene therapies have been approved for use, including treatments for severe combined immunodeficiency. Researchers are working on gene therapies for conditions such as cystic fibrosis, amyotrophic lateral sclerosis, age-related macular degeneration and sickle cell anaemia, among others.
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In recent years, gene therapy has been expanded to cancer treatment, and has been approved in the US for leukaemia.
Gene therapy involves inserting whole copies of genes into cells, rather than changing the genes that exist inside cells, which makes it different from gene editing.