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Helen Obando, a shy slip of a girl, lay curled in a hospital bed in June waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest.
The hope was that the treatment would cure her of sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and early death.
Sedated with Benadryl to prevent an allergic reaction to the garlicky-smelling preservative in the drip, Helen, who at 16 was the youngest person ever to undergo the therapy, was sound asleep for the big moment.
“Wake up,” her younger brother, Ryan, said, shaking her leg so she could push the button to start the drip. But she could not be roused, so he pushed it himself.
news & eventsBoston Children’s Hospital receives grant for sickle cell disease researchThe Bill and Melinda Gates Foundation aw...
news & eventsStatement on NHLBI Decision to Pause the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Si...bluebird bio, Inc. suspended its clinica...
news & eventsFDA Clears Graphite Bio to Begin Trial for Gene Therapy in Sickle Cell DiseaseIn response to Graphite Bio’s investig...
news & eventsEarly clinical trial data show gene therapy reversing sickle cell anemiaAfter over a decade of preclin...
news & eventsCIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell DiseaseThe California Institute for Regenerativ...
videos & visualsGene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
education & researchBiologic and Clinical Efficacy of LentiGlobin for Sickle Cell DiseaseBackground: Sickle cell disease is char...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.