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Sickle Cell Breakthrough
Embryonic
stem cells could hold the key.
Sickle cell anaemia is a condition that mainly affects people
of African and Caribbean descent.
People who have the sickle cell gene
develop sickle-shaped cells in their blood which are poor oxygen carriers
and clog up the blood vessels. This can cause severe anaemia, accompanied
by pain and a propensity to developing infections, and can also lead
to strokes and kidney, lung and heart problems.
While serious episodes can be controlled with blood transfusion the only
cure is a bone marrow transplant from a matched donor.
New research by a team from the University of California suggests that
in future it may be possible to correct the sickle defect with stem cells.
Using embryos from genetically-engineered mice carrying the human sickle cell mutation, stem cells were extracted and the defective gene was replaced with a healthy copy. Every stem cell generated from the original then became corrected.
It is also thought that the same stem cell therapy
could be used to cure another genetic blood disorder - thalassaemia.
Most common among Mediterranean people this anaemic condition is caused
by a different blood cell abnormality.
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