The Scientist : Gene therapy for sickle cell disease [18/12/2001]

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by Tudor Toma

RESEARCH ROUND-UP

Gene therapy for sickle cell disease

Email: Tudor Toma - t.toma@ic.ac.uk
News from The Scientist 2001, 2(1):20011218-03

Published

18 December 2001

Sickle cell disease (SCD) is caused by the homozygous inheritance of a single point mutation in the β globin gene (hemoglobin S) that manifests itself as an anemia with characteristic sickle-shaped red blood cells. Currently available treatments are only symptomatic, but in December 14 Science, Robert Pawliuk and colleagues from Massachusetts Institute of Technology, Cambridge, US show evidence that it is possible to correct sickle cell disease in transgenic mouse models using gene therapy.

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