Sickle-cell anaemia is an autosomal recessive disorder that causes the sufferer’s red blood cells to change from its regular oblong shape into a sickled form. Sickle-cell anaemia is caused by a mutation in the haemoglobin gene. As a result of this mutation, the abnormality of the shape of the cells obstructs blood flow and oxygen flow into a tissue or organ, which can result in a shorter life-expectancy in affected individuals.
A gene therapy experiment carried out on mice by the Massachusetts Institute of Technology attempted to replace the sickle-cell encoding mutation with an anti-sickling gene using stem cells.
Red blood cells are formed from the stem cells in bone marrow. Therefore the treatment had to be delivered through that pathway. Firstly, the bone marrow of the mice was killed will irradiation, before the treatment was applied.
The way in which this was carried out was by delivering the anti-sickling gene into the blood forming stem cells of the affected mice. This new genetic matter is then taken up by the stem cells in the bone marrow which in turn give rise to new red blood cells. These newly formed red blood cells now have been genetically modified to exhibit normal red blood cell structure.
Prolonged monitoring of the treated mice found that the instances of sickle-cell anaemia had dropped. Some tests proved that it had been eradicated or completely eliminated.
Whilst the treatment proved effective on the mice in eradicating the instances of sickle cell, further research will continue to be carried out to develop a safe method for administering this gene therapy in humans. Based on the evidence, this cure is not too far away.
By Kimberley Martinz s4201269
Original Article:
http://www.sciencemag.org/cgi/content/full/sci;294/5550/2368?maxtoshow=&HITS=10&hits=10&RESULTFORMAT=&fulltext=sickle+cell+&searchid=1&FIRSTINDEX=0&resourcetype=HWCIT
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