Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
by Nancy Fliesler,Children's Hospital BostonNew work suggests ways to target the BCL11A protein with drugs, enabling patients with sickle cell disease to produce non-sickling fetal hemoglobi
Updated on: January 17,2025
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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
by Nancy Fliesler,Children's Hospital BostonNew work suggests ways to target the BCL11A protein with drugs, enabling patients with sickle cell disease to produce non-sickling fetal hemoglobi
Updated on:January 17,2025
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