Stem cells treat babies before birth
Wednesday, 30 January 2008
University of Queensland
The extraordinary results of an in utero stem cell study could lead
to a new treatment for babies with brittle bones, as well as a range
of other disabling conditions, according to a maternal-fetal medicine
researcher, now based at The University of Queensland (UQ).
Action Medical Research has announced the outcomes of an Imperial
College London study, conducted by a team led by Professor Nicholas
Fisk, that could lead to a stem cell treatment for babies with
brittle bones - before they are even born.
Professor Fisk, who now heads the new $66m UQ Centre for Clinical
Research, said the work held potential for improving treatment of
other disabling conditions such as muscular dystrophy and congenital
brain diseases.
Brittle bone disease or Osteogenesis imperfecta (OI), as the
inherited disease is known, affects babies whilst they are inside
their mother's womb. This is because collagen, one of the main
building blocks for bone, fails to develop properly. The disease is
detected by DNA testing or ultrasound before birth and leads to weak
bones and stunted growth.
The team, led by Professor Nicholas Fisk, transplanted specially
manipulated stem cells into 14 day old mouse fetuses that had OI.
These mice had a reduction in long bone fractures of two thirds,
compared with an untreated group, by the time they were twelve weeks
old. They also found that the bones of these mice were stronger,
thicker and longer than those with the disease that had not received
the transplants.
These outstanding results, published in the journal Blood, suggest
that, with further research, this treatment could be translated to
human babies in pregnancies that are affected by OI.
Dr Yolande Harley of charity Action Medical Research, which funded
the project, said:
"Professor Fisk's work is a real breakthrough. It suggests that if
stem cells could be successfully transplanted before a baby with OI
is even born, it could mean a significant improvement in the child's
health and quality of life.
"This is a tremendous piece of work that could have implications for
many more diseases and conditions," he said
Professor Fisk said the research had shown "a profound therapeutic
benefit" of intrauterine stem cell therapy.
"It has significance not only for treating this and other disabling
conditions in affected fetuses inside the womb, but also for future
related work. It will help us to understand what it is that leads to
such a marked effect after a single transplant of stem cells, so that
this can be harnessed to improve the results of stem cell therapy in
repairing adult tissues and degenerative conditions.
"Our work suggests that, in the future, it could be possible to take
stem cells from an unborn baby carrying the abnormal OI gene,
manipulate them to correct the errant gene and then put them back
into the fetus to allow it to develop properly".
http://www.sciencea
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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