Treating Sickle Cell Disease

New Scientist reported that switching off a certain gene could treat sickle cell disease.  The disease creates

[caption id="attachment_3761" align="alignleft" width="200" caption="Silenced gene. Turning off a gene called BCL11A in mice with sickle cell (right) disease helps them to produce red blood cells (left) with working hemoglobin molecules. Credit: Fotosearch"][/caption]

mutated blood cells which contain long sticky chains.  The mutated cells could clog small vessels, cutting off oxygen to organs.  The gene scientists are targeting is called BCL11A. The gene switches fetal haemoglobin to adult haemoglobin.  Studies have been conducted to turn off the BCL11A gene in mice.  By doing so this allows the mice to produce fetal blood cells with working hemoglobin molecules.  Harvard Medical School conducted an experiment to switch of the BCL11A gene in mice with sickle cell disease.  After doing so it was observed that the mice produced 20 times more fetal haemoglobins, the cells produced contained nearly zero sickle cells.  Organs in the mice were virtually completely healthy.  The article goes on to explain that the gene could be targeted in humans by redesigning the length of the patients RNA and injecting it into the blood stream.  The drug hydroxyurea could also be taken to produce fetal haemoglobin.  The problem with redesigning RNA is the expense.  On the other hand hydroxyurea has been known to reduce white blood cells.  Science Now reported identical results from the Harvard experiment.  Science Now explains that more complications would arise if the gene was targeted in humans rather than mice.  All in all more research has to be performed before targeting the BCL11A gene in human beings.  Perhaps within the next few years scientists will have a better method to targeting the gene and individuals with sickle cell disease will be cured.