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Universal treatment for rare diseases? Cure for sickle cell, immune deficiencies created from umbilical cord blood

[I]nfusing umbilical cord blood — a readily available source of stem cells — safely and effectively treated 44 children born ...
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CRISPR therapy demonstrates ‘real curative potential’ for sickle cell disease, beta thalassemia

The first two patients to receive a CRISPR-based treatment for the inherited blood disorders sickle cell disease and beta thalassemia ...
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Podcast: Has sickle cell disease met its match in CRISPR gene editing?

Dr. Brenda Eustace, director of discovery research at Vertex, joins plant geneticist Kevin Folta to discuss this promising treatment ...
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