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 The technique, developed with the technology commercialization firm
Imperial Innovations, replaces the defective gene behind the
inherited lung disease by using inhaled molecules of DNA to deliver
a normal working copy of the gene to lung cells.
"Patients who received the gene therapy showed a significant, if
modest, benefit in tests of lung function compared with the placebo
group," said Eric Alton, a professor at Imperial College London who
led the trial.
Briefing reporters about the results, he said they were
"encouraging" but cautioned that because the effect was
inconsistent, with some patients responding better than others, the
gene therapy was not ready for regular clinical use.
"The effects were modest and variable," he stressed.
Cystic fibrosis is the world's most common lethal inherited disease,
affecting more than 90,000 people globally. Patients' lungs become
filled with thick sticky mucus and they are vulnerable to recurrent
chest infections, which eventually destroy the lungs.
In this trial, results of which were published on Friday in The
Lancet Respiratory Medicine journal, 136 CF patients aged 12 and
over received monthly doses of either the gene therapy or a placebo
for one year.
Patients who received therapy had significant, if modest, benefit in
lung function compared with placebo, making the trial the first in
the world to show that repeated doses of gene therapy can have a
meaningful effect on cystic fibrosis.
Dominic Wells, a professor of translational medicine at Britain's
Royal Veterinary College who was not involved in the trial, said its
findings laid "a strong foundation for the development of more
potent gene transfer systems".
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In this trial, patients were treated by inhaling molecules of DNA
wrapped in fat globules, or liposomes, that deliver the gene into
the cells in the lung lining.
Alton said his team is now planning follow-up studies to look at
whether higher, more frequent doses might have a greater benefit,
and to test ways of combining the gene therapy with drug treatments
to potentially boost its effect.
The team has also developed another gene therapy delivery method
using a hybrid virus created in the lab by combining a so-called
lentivirus with another called Sendai virus, which is known for its
ability to efficiently deliver replacement genes to the intended
target.
(Reporting by Kate Kelland; Editing by Mark Heinrich)
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