Scientists
eliminated HIV from the entire genome of lab mice for the first time ever using
a slow-acting drug and gene-editing.The feat,
revealed in a publication today, suggests this two-pronged technique could be
the basis for the first universal cure in humans, with human clinical trials
slated to start next summer.
Only two
people have been cured of HIV, both had terminal blood cancer and underwent a
risky bone marrow transplant that obliterated both diseases.But the
transplant technique has not worked in anyone else - proving fatal in some -
and it effectively requires that the patient have both HIV and cancer.
Now, a team
spearheaded by an HIV expert in Nebraska and a gene-editing expert in
Philadelphia has presented the unprecedented fruits of a five-year project:
using a slow-acting drug called LASER ART that corners the virus, followed by
CRISPR Cas9 gene-editing that blitzes it.
In a new
paper, they reveal this approach successfully eliminated HIV from the entire
genome of a third of their lab mice.
HIV is so
hard to obliterate because it is a virus that infects the genome. It buries
itself inside hidden reservoirs, ready to mount a resurgence at any point. A
team between Philadelphia and Nebraska has shown they were able to eliminate
the virus even from hard-to-access reservoirs
Even they
were surprised.
'We didn't believe it,' Dr Howard E Gendelman, Director of the Center for Neurodegenerative Diseases at the University of Nebraska Medical Center, told DailyMail.com.
'We thought
it was a fluke, a problem with the graphs; that the cells carrying HIV had
died; that our assay system was wrong.'It was only
after we repeated it a couple of different times,' he says, that they accepted
they had hit the veritable jackpot.What's more,
journals didn't believe it.
'After we
got it right, we submitted it for publication and it was rejected from many
different journals,' Dr Gendelman said.'They had a
hard time believing HIV could be cured.'
He and his
co-author (and 'best bud') Dr Kamel Khalili, of Temple University in
Philadelphia, added no less than 20 supplemental figures to their paper - far
more than usual - to prove that their results were not a fluke, finally getting
the green light from Nature Communications.
'There was a
lot of frustration, self-introspection, denials, reaffirmation, and just
laborious day by day activities to prove it,' Dr Gendelman said.
HIV is so
hard to obliterate because it is a virus that infects the genome.It buries
itself inside hidden reservoirs, ready to mount a resurgence at any point.
These days,
we have incredibly effective drugs (called ART, or anti-retroviral therapy)
that suppress the virus to such an extent that it is undetectable, and cannot
be transmitted to another person.
It means
people who have HIV can live a long, healthy life without the virus turning
into AIDS.
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