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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.
The Wellcome Trust and other UK funders declared their full support for genome editing research. They are upbeat about future therapies. For the large part, that means editing the genomes of affected cells and tissues, to alleviate damaged lungs, wasted muscles, blood disorders and the like. But genome editing opens the door to more controversial therapies: ones that prevent disease by altering human embryos.
Other procedures can already prevent genetic diseases from being passed on. Pre-implantation genetic diagnosis is routinely used for couples who know they are at risk of passing on a genetic disorder. But the funders are right to call for a national debate. Genome editing has immense potential. It is hard to see how its safety could be proven in human embryos without risk, but the same was true for IVF in the 1970s. For some cases, embryo editing may be morally justified.
Read full, original post: The Guardian view on human genome editing: find, replace – and cure