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Stem-cell therapies for blood diseases - FREE ACCESS

Claudio Bordignon

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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• In a unique discovery, scientists have demonstrated that undifferentiated human stem cells can travel down a developmental pathway and become blood cells.
Researchers Walter E. Goldstein and Warren K. Miller have developed a special procedure that actually turns stem cells into red blood cells.

“For the first time in history, doctors will be able to obtain and transfuse disease-free bioreactor-produced universal blood properly matched to patient needs”, Goldstein said. Moreover, the ability to create blood in the lab has obvious implications for augmenting human blood supplies around the world to treat cancers of the blood and bone marrow (such as leukemia and myeloma).

Goldstein, Coordinator of the Biotechnology Center at UNLV, co-developed the technology prior to coming to UNLV; but he hopes to bring their discovery into the UNLV Biotech Center for development to commercialization.
The researchers emphasize that while the work shows great promise towards taming stem cells, it faces many challenges and remains basic science. Its use in transplants and transfusions may be years away. However, navigating the clinical trial world and gaining FDA approval could happen as early as 2015.

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