Gene Therapy

Study results from L. Lisowski and colleagues update understanding of gene therapy

Published in Medical Device Business Week, May 28th, 2008

"beta-Thalassaemia major is a congenital anaemia for which there is presently no curative therapy other than allogeneic haematopoietic stem cell transplantation. This therapeutic option, however, is not available to most subjects for whom there is no available human leucocyte antigen-matched bone marrow donor," investigators in the United States report.

"The transfer of a regulated globin gene in autologous haematopoietic stem cells is therefore a direly needed alternative treatment. This strategy, simple in principle, raises major challenges in terms of controlling transgene expression, which ideally should be erythroid-specific, differentiation- and stage-restricted,...

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Source: Medical Device Business Week (2008-05-28)

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