In a race to cure his daughter, a Google programmer enters the world of hyper-personalized drugs.
To create atipeksen, he borrowed from recent biotech successes like gene therapy. Some new drugs, including cancer therapies, treat disease by directly manipulating genetic information inside a patient’s cells. Now doctors like Yu find they can alter those treatments as if they were digital programs. Change the code, reprogram the drug, and there’s a chance of treating many genetic diseases, even those as unusual as Ipek’s.
That technology is called ‘antisense.’ Inside a cell, DNA encodes information to make proteins. Between the DNA and the protein, though, come messenger molecules called RNA that ferry the gene information out of the nucleus. Think of antisense as mirror-image molecules that stick to specific RNA messages, letter for letter, blocking them from being made into proteins. It’s possible to silence a gene this way, and sometimes to overcome errors, too.
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