NCCS scientists are studying the use of cord-lining stem cells to transport a specific gene into the body that will then go on to producing a clotting protein known as factor VIII, which patients lack
Haemophilia A is a rare genetic
condition in which the blood does
not clot properly.
Local scientists are studying the
use of cord-lining stem cells to
transport a specific gene into the
body that will then go on to produce
a clotting protein known as
factor VIII, which patients lack.
In animal studies led by Professor
Kon Oi Lian of the National Cancer Centre Singapore, stem
cells carrying factor VIII were introduced
into mice with the condition.
Scientists found that the animals
began to produce the protein.
The mice to which the gene was
introduced also bled less when
their tails were clipped.
“We were able to show, at least in
a small animal model, that these
cord-lining epithelial cells not only
secreted factor VIII, but were able
to mitigate, though not cure, the
haemophilic mice,” said Prof Kon.
(AAVs), which are not known to
cause disease in humans, are now
the commonly used vector for
transporting the gene, but there
have been some challenges.
One problem is that people infected
before by the virus already produce
against it, which means they would
not beable to receive the gene.
“The AAV also does not insert itself
into the genome, so it is possible
the effect will wear off after a
while,” added Prof Kon, who is also
with the Yong Loo Lin School of
Medicine. Other viral vectors studied
that insert themselves into
the patient’s genome have had adverse
Prof Kon noted that cord-lining
stem cells could provide an alternative
method. Her team started
studies on dogs last year.