2016
Gene therapy for sickle cell disease
Abstract: No randomised or quasi-randomised clinical trials of gene therapy for sickle cell disease were reported. Thus, no objective conclusions or recommendations in practice can be made on gene therapy for sickle cell disease. This systematic review has identified the need for well-designed, randomised controlled trials to assess the benefits and risks of gene therapy for sickle cell disease.
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Sickle cell disease (SCD) is a genetic disorder found mostly in people of African descent. The inherited blood disorder affects the shape of the red blood cells and marked commonly by haemoglobin S (HbS) as opposed to the normal haemoglobin (HbA) (Matthie, 2013;Olowoyeye & Okwundu, 2010). It is estimated that SCD has an impact on approximately 20-25 million individuals globally with most residing in sub-Saharan Africa.
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confidence: 99%