http://www.bloomberg.com/news/2015-...e-drives-hearing-loss-drug-search-health.html
The therapy uses a disabled cold-causing virus that enters so-called supporting cells in the inner ear, where it delivers its payload -- a gene called atonal-1 that tells the cells to grow into hair cells. Scientists have been working for decades on using genes to treat maladies including cancer, HIV and blindness, with mixed results.
A trial more than a decade ago of an experimental gene therapy cured 17 infants with a rare immune disorder, but caused leukemia in five patients, and killed one. Trial results from a second study, published last year, have shown no signs of leukemia.
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Getting Novartis’s hearing-loss drug into a patient’s inner ear may prove to be the trickiest part of the treatment, according to Fishman. A surgeon must drill a tiny hole in a bone called the stapes, then inject the drug with a needle. It won’t help patients with genetic hearing loss.
The therapy uses a disabled cold-causing virus that enters so-called supporting cells in the inner ear, where it delivers its payload -- a gene called atonal-1 that tells the cells to grow into hair cells. Scientists have been working for decades on using genes to treat maladies including cancer, HIV and blindness, with mixed results.
A trial more than a decade ago of an experimental gene therapy cured 17 infants with a rare immune disorder, but caused leukemia in five patients, and killed one. Trial results from a second study, published last year, have shown no signs of leukemia.
...
Getting Novartis’s hearing-loss drug into a patient’s inner ear may prove to be the trickiest part of the treatment, according to Fishman. A surgeon must drill a tiny hole in a bone called the stapes, then inject the drug with a needle. It won’t help patients with genetic hearing loss.