Professor Adrian Thrasher, of the UCL Institute of Child Health/Great Ormond Street Hospital, has welcomed a French-led study showing the success of gene therapy in a case of beta thalassaemia. (Nature, 16 Sept 2010)
“This is an encouraging study showing proof of principle that gene therapy can have genuine therapeutic effects in another haematological disorder, this time beta thalassaemia. Beta thalassaemia and diseases like it are relatively common single gene disorders, worldwide.
However, this study also highlights a need to design vectors – the viruses that carry the new gene into the blood cells - that are less likely to disturb function of other genes (and potentially cause side effects). We also need to develop transplant procedures that allow the establishment of normal blood cell devlopment from the gene corrected cells. In this case a single cell is effectively driving the clinical effect, whereas normally one would expect many many more.
This underpins the need for the research we are doing and would want to do in these areas.”
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