The Therapeutic Promise of Genome Editing: In My Lifetime?

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Genome editing has potential to advance human health with several different approaches.

By T. Oliver Chao, Ph.D., Sanofi

 

When a friend decided to leave the pharmaceutical industry two years ago, he asked me half-heartedly what to look out for in the future. Without thinking too hard or long, I answered, “Remember the name CRISPR! It may save your life later.“ That might have been an overstatement then, as my friend has grown into adulthood without any apparent genetic disorder or other visible life-threatening illness. Now as we march into mid-2016, CRISPR’s magic wand seems capable of touching a wide spectrum of human diseases—from predisposed genetic liability to epigenomic modification and from metabolic disorders to infectious pathogens. Perhaps my friendly prediction two years ago was not too far off after all. It is probably safe to say that CRISPR is on almost every health-conscious person’s mind nowadays. From New York Times special articles to late-night TV talk shows, from a highly anticipated (wishful) confirmation of last year’s Nobel Prize to a 12-figured 2016 calendar by Nature Protocols, CRISPR/Cas seems to have become the promised innovation destined to change our lives. Or, according to the U.S. Director of National Intelligence, a potential “weapon of mass destruction and proliferation.” Anyway, it is probably safe to say that CRISPR is no longer just a geek topic.

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May 2016

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