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After decades of neglect, sickle cell disease could soon be transformed with gene-based therapies

Por: The Boston Globe Business June 11, 2023

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It’s one of the great paradoxes of medicine.Sickle cell disease was the first human disorder understood on a molecular level, its underpinnings explained in a landmark 1949 paper by a future two-time Nobel laureate.Yet the inherited blood disease ― which causes unpredictable bouts of crushing pain, damages organs, and cuts lives short ― has eluded transformative treatments longer than a number of other genetic diseases, including far rarer... + full article



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