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>> Haemophilia B is a
rare bleeding disorder.
It affects around
one in 30,000 males.
There's a problem
with blood clotting.
In patients with haemophilia,
they lack a protein
called Factor IX.
Factor IX is a protein
that circulates in blood
and when we cut ourselves it
starts the coagulation of blood
to protect us from
bleeding excessively.
Now, in patients
with haemophilia,
the gene that's responsible for
making factor IX is damaged.
>> There is currently a therapy.
Patients can be given
recombinant protein,
but it has to be
given very frequently.
It's extremely expensive
and therefore not readily
available throughout the world
and it's a very invasive
process.
So, it is a disease that's
in need of better treatment.
>> One way of treating
this condition is
to replace the damaged
gene with a normal copy
of fully functional gene.
And so the way we've decided to
deliver this gene to the liver
which is where factor IX is
usually made is to use viruses.
We've taken out the properties
of the virus which is important
for causing disease and replaced
it with the factor IX gene
and so what we're doing
essentially is using the virus
as a vehicle to deliver
our gene to the liver.
In the incubators are cells
that we use to make the virus.
These are our production
line for making the vector.
When we treat patients,
we infuse it directly
into the patient's vein
and that's essentially it;
that's the treatment done.
>> It is impressing
that literally somebody
is injecting cells
that making factor IX into
a vector that is a virus.
When I cut myself shaving for
example, the bleeding just stops
which wasn't happening
before and I feel better
in general I don't have
small bleeds anymore,
bruises and stuff like that.
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>> The collaboration
between St. Jude
and University College London
has really been critical
to the success of this project.
>> We've been able to
develop this project together
and accelerate its
passage to clinic.
Before it can used by the
wider haemophilia community,
it needs to go through
several other steps to show
that the safety aspects of
this treatment are robust
and long lasting.
And for this treatment
to become available,
it may take another
five to ten years.
>> We're also interested in
opening this project to children
who may in fact benefit more
than adults from this approach.
>> I think the real
take on this research is
that it has a potential to
revolutionise the lifestyle
of these patients, to
revolutionise haemophilia care.
>> This kind of a solution
is something permanent
that can make a difference
for a lot of people.
>> If we can make it
work for haemophilia,
then there is every hope that we
can make it work for a variety
of other disorders including
diabetes, including cancer.
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