Targeting DNA

[TECH NEWS]

After 20 years of high-profile failure, gene therapy is finally well on its way to clinical approval. The concept is simple: if a mutated gene is causing a problem, replace or supplement it with a new, accurate copy. In theory, such a strategy could not just treat, but cure countless human genetic diseases. In practice, however, developing safe and effective gene therapies has not been easy. But more and more researchers are convinced that the technique is on the brink of becoming a common medical practice. “It’s an incredibly exciting time for the field,” says researcher and medical oncologist David Kirn, founder, president, and chief medical officer at Jennerex, Inc., a San Francisco-based biotherapeutics company that develops and commercializes oncolytic drugs. In the last year alone, he says, major breakthroughs have been published for the use of gene therapy in patients with hemophilia, solid tumors, and leukemia, not to mention the dozens of trials yielding positive results for gene therapies to treat various types of blindness.

June 1, 2012, Jef Akst / The Scientist