For decades, transplantation of haematopoietic stem cells — either unmodified, or genetically modified to correct genetic disorders — has been used to treat disorders of the blood and immune systems. The present challenge is to reduce the risk of such transplants and increase the number of patients who can safely access this treatment. In developing countries, such ‘one-shot’ treatments are highly desirable because chronic treatments are difficult to sustain. To make these therapies more accessible and effective it will be important to improve clinical protocols and gene-delivery vectors, and to gain a deeper understanding of stem cells.
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The author is indebted to L. Reiss and M. Cassin for the preparation of the manuscript.
Claudio Bordignon acts as a consultant for MolMed SpA.
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Bordignon, C. Stem-cell therapies for blood diseases. Nature 441, 1100–1102 (2006). https://doi.org/10.1038/nature04962
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