The Corpus Callosum

only that, but the genetically modified “organism” is an engineered
HIV.  At least, that is what researchers at Emory College in
London are contemplating.

Aids virus could provide cure for cystic fibrosis

Henderson, Science Editor

19, 2007

The Times

modified version of the virus that causes Aids could become the basis
of a prenatal gene therapy for cystic fibrosis, new research has

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.

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.

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

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.  


  1. 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.

  2. #2 cystic fibrosis treatment
    March 15, 2010

    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.

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