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Wednesday, November 01, 2006

The Glimmering Promise of Gene Therapy

Its history is marred by failures, false hopes, and even death, but for a number of the most horrendous human diseases, gene therapy still holds the promise of a cure. Now, for the first time, there is reason to believe that it is actually working.

By Horace Freeland Judson

Credit: Illustration by Chris Buzelli

By the late 1960s, molecular biologists had erected an overarching explanation of how genes work--their substance, their structure, their replication, their expression, their regulation or control. Or at least they had done so in outline, for prokaryotes, the simplest single-celled organisms (which include bacteria), and for the viruses, called bacteriophages, that prey upon them. The leaders of the field were now looking to a far more difficult problem: doing it all over again for higher organisms.

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