The heritable blood disorder b-thalassaemia is common in Mediterranean countries and in much of Asia - in the worst-affected places, such as Cyprus, as many as one in seven people carry the disease-causing mutation. At present, patients can be treated only with regular blood transfusions, iron-purging drugs or donor bone-marrow transplants. Because it is caused by the lack of a single protein molecule, this condition is high on the list of human diseases that might be tackled with gene therapy. Attempts to validate gene therapy approaches for tackling the disease in animal models have, however, been thwarted by insufficient expression of the necessary globin molecule.
This week [Nature, Vol. 406, Issue 6791, July 6, 2000], Michel Sadelain of the Memorial Sloan-Kettering Cancer Center, New York, and colleagues report that they have solved this problem in a mouse b-thalassaemia model. They used recombinant lentiviruses to transfer the human b-haemoglobin gene, and its locus control region, into mouse blood (or haematopoietic) stem cells. The resulting bone marrow cells are stable and produce haemoglobin at potentially therapeutic levels when reconstituted into mice. This technique also provides a proof-of-principle for analogous stem-cell therapies.
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(C) Nature press release.
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