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Cambridge, UK – Scientists have discovered a way to significantly enhance the effectiveness of blood stem cell gene therapy. By repurposing a cancer drug, they’ve tripled the lifespan of these crucial cells outside the body, a critical step in treating life-threatening blood disorders.
Blood stem cells are the body’s master cells, responsible for producing trillions of blood cells daily. Gene therapy, while a lifeline for millions with blood cancers and genetic diseases, has been hampered by the rapid decline of blood stem cells when removed from the body for treatment.
Researchers at the University of Cambridge have identified the root cause of this decline – a severe stress response triggered when stem cells are placed in a laboratory dish. By administering the cancer drug ruxolitinib, they managed to mitigate this stress response, dramatically improving the cells’ viability.
This breakthrough could revolutionize gene therapy by increasing the number of high-quality stem cells available for transplantation, leading to better outcomes for patients. The study is published in the Blood Journal.
Professor Elisa Laurenti said, “This discovery is a game-changer. We can now potentially improve the safety and efficacy of gene therapy for countless patients. It’s an exciting step forward in harnessing the power of blood stem cells to treat a wide range of diseases.”
Dr. Carys Johnson added, “Our findings highlight the critical importance of understanding the challenges faced by blood stem cells outside the body. By overcoming these hurdles, we can unlock their full potential for therapeutic applications.”
The research team is now working to incorporate ruxolitinib into standard gene therapy protocols, with the aim of bringing this enhanced treatment to patients as soon as possible.
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Citation:
New way to extend ‘shelf life’ of blood stem cells can improve gene therapy (2024, August 15)
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