Nonsense mutations: running the red light
C Ainsworth - Nature, 2005 - go.gale.com
C Ainsworth
Nature, 2005•go.gale.comOn 28 November, two patients in an Israeli hospital downed a vanilla-flavoured shake in the
hope of curing their cystic fibrosis. The drug in the shake is meant to get their cells to ignore
the genetic mutation that makes their lives a misery. Remarkably, if this trial works, the very
same drug might be used to help patients with a huge range of genetic diseases.At the
moment treatments for cystic fibrosis concentrate on the condition's symptoms, such as lung-
clogging mucus, rather than its underlying cause--a faulty gene. Advocates of gene therapy …
hope of curing their cystic fibrosis. The drug in the shake is meant to get their cells to ignore
the genetic mutation that makes their lives a misery. Remarkably, if this trial works, the very
same drug might be used to help patients with a huge range of genetic diseases.At the
moment treatments for cystic fibrosis concentrate on the condition's symptoms, such as lung-
clogging mucus, rather than its underlying cause--a faulty gene. Advocates of gene therapy …
On 28 November, two patients in an Israeli hospital downed a vanilla-flavoured shake in the hope of curing their cystic fibrosis. The drug in the shake is meant to get their cells to ignore the genetic mutation that makes their lives a misery. Remarkably, if this trial works, the very same drug might be used to help patients with a huge range of genetic diseases.
At the moment treatments for cystic fibrosis concentrate on the condition's symptoms, such as lung-clogging mucus, rather than its underlying cause--a faulty gene. Advocates of gene therapy aim to correct this by delivering working copies of the cystic fibrosis gene to a patient's cells, but results have been mixed so far.
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