New Gene Therapy

Abstract

Modern medicine has advanced tremendously in the 20th century, yet many people still die each year from conditions like heart attacks, cancer, and infectious diseases. Genome editing looks set to transform clinical medicine over the next few decades, because it now makes it possible to alter genes either in a living person, or in an infectious agent like a virus. One particular type of disease where genome editing is likely to have a big impact is single gene disorders such as cystic fibrosis, Huntington’s disorder, and muscular dystrophy. Because these disorders are due to a mutation in a single gene, there is hope that it should be possible to use genome editing to ‘correct’ the mutation in the cells of a sufferer. But genome editing also offers much promise for the treatment of more common disorders such as cancer. In the latter case, a mutated oncogene could be corrected by genome editing, or this approach used to enhance the ability of special immune cells in the body that target cancer cells. Genome editing also has much potential for targeting viruses like as HIV. In this case, there are two possible approaches. One involves targeting the HIV genome sequence, the other involves creating a mutation in a gene coding for a human protein that HIV uses to get inside an infected person’s cells. Bacterial infections might also one day be targeted by genome editing. However, many practical obstacles remain, the main one being how to get genome editing tools into a person safely.