Gene therapies treat or prevent disease by tweaking the content or expression of cells' DNA.
Gene therapy has been headline news in recent years, in part due to the rapid development of biotechnology that enables doctors to administer such treatments. Broadly, gene therapies are techniques used to treat or prevent disease by tweaking the content or expression of cells' DNA, often by replacing faulty genes with functional ones.
Although there are mitochondrial diseases that could someday be cured with gene therapy, currently, the term gene therapy refers to treatments that target nuclear genes — the genes on the 23 pairs of chromosomes inside the nucleus. In vivo gene therapy means that the gene therapy itself is injected or infused into the person. This can be through injection directly to the anatomic site where the gene therapy is needed , or it can mean injection or infusion of a genetic payload that must travel to the body tissues where it is needed.
After moving away from retroviruses, researchers turned to adenoviruses, which offered the advantage of delivering the genetic payload as an episome — a piece of DNA that functions as a gene inside the nucleus but remains a separate entity from the chromosomes. The risk for cancer was extremely low with this innovation, but adenovirus vectors turned out to stimulate the immune system in very powerful ways.
Gene therapy has also been useful in treating hereditary retinal diseases , for which other treatments have not been useful.
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