Disabled Aids virus could provide cure for cystic fibrosis
Mark Henderson, Science Editor
March 19, 2007
From The Times
A modified version of the virus that causes Aids could become the basis of a prenatal gene therapy for cystic fibrosis, new research has suggested.
British scientists have discovered that a form of HIV, adapted to be harmless, is a promising vector for ferrying replacement genes into the lungs of affected foetuses. Experiments at University College London have already shown that the modified virus can infect the lung tissue of unborn mouse pups, suggesting it could be used to correct the genetic defects that cause cystic fibrosis.
The virus could potentially be engineered to carry working copies of the gene which, when it goes wrong, causes the devastating lung condition. It would then be used to infect foetuses known to be affected by the disease before they were born.
The idea is conceptually simple. The virus carries the correct gene to the tissue where the defective gene is active. The correct gene then takes over, at least partly, and either cures the disease or diminishes the severity.
Naturally there are many technical problems. Ideally, the virus would selectively go to the tissue where it is needed. That is done by modifying the surface proteins of the HIV to more closely match those of influenza virus. Then you have to get the correct gene, and get it to go where it is supposed to go, and get it to take over the function of the disease-causing gene. All without doing more harm than good.
It occurs to me that some people may object to the idea of engineering viruses in this way. Others may lack confidence that something derived from HIV could be rendered nonpathogenic. Others may object to the notion of deliberately infecting unborn fetuses with anything, or even intervening medically in any way. Others may raise the issue of informed consent, as fetuses cannot be informed, and cannot consent. Even allowing for the generally-held notion that parents can make these decisions, one might wonder how well the average parent could understand what is being proposed well enough to give truly informed consent.
What this shows is that there are numerous challenges, both technical and ethical, before such research could proceed with humans.
If the technical problems are overcome, but not the ethical ones, we would at least have the ability to treat cystic fibrosis in mice.
The London group is collaborating with the Autralian group that developed a mouse model of cystic fibrosis. Similar work is being done in Singapore, with monkeys and hemophilia.
Comments
Hi
I'm wondering if there is a follow up on this post? Gene therapy seems very promising but hasn't proven to be effective yet. Hopefully 1 day it will be.
Posted by: Find out what cystic fibrosis is | March 2, 2009 1:02 PM
How do chloride ions affect water transport and mucus buildup in cystic fibrosis? In the disease cystic fibrosis there is a defect in the ability of endothelial cells to transport Cl- ions.
Posted by: cystic fibrosis treatment | March 15, 2010 8:14 AM