After decades of neglect, sickle cell disease could soon be transformed with gene-based therapies
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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…
This story from bostonglobe.com was posted on 2023-06-11 by @starfly.