The Guardian view on human genome editing: find, replace – and cure | Editorial
More than 10,000 diseases result from mutations in one or other of the 20,000 genes in the human genome. The disorders are rare. But across the world they affect millions of people. They are responsible for a heavy loss of life. Scientists have worked for decades to perfect gene therapies that might treat some of these conditions. The latest weapon in their armoury is a technique called Crispr-Cas9, a form of genome editing. It works like the find and replace function on a word processor. Instead of correcting clumsy typos, it cuts out faulty DNA and replaces it with the right genetic lettering.
Researchers are using Crispr-Cas9 to learn how genes control development, and how they affect health and disease. Radical therapies are on the cards. Scientists hope to draw immune cells from cancer patients, and rewrite their genomes, so that when the cells are put back they destroy the patients' tumours. Separately, trials are under way to make people resistant to HIV by writing natural immunity into the DNA of their blood cells. It is early days, but the medical potential is breathtaking.
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