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At 16, She’s a Pioneer in the Fight to Cure Sickle Cell Disease
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.
education & researchGene Therapy: An Experimental Technique to Treat Genetic DiseasesGene therapy is an experimental techniqu...
videos & visualsAlabama Man Free of Sickle Cell After Gene Therapyhttps://www.youtube.com/watch?v=E9vYON8D...
news & eventsSCDAA News Advisory: Partial Hold on Gene Therapy TrialOn December 20, the FDA placed a partial...
news & eventsGene therapy may help cure sickle cell disease, study saysA gene therapy that could provide a perm...
education & researchBiologic and Clinical Efficacy of LentiGlobin for Sickle Cell DiseaseBackground: Sickle cell disease is char...
news & eventsBluebird Bio Presents Data From LentiGlobin Gene Therapy TrialBluebird Bio’s investigational gene th...
news & eventsExperimental Gene Therapy Reverses Sickle Cell Disease for YearsA study of an investigational gene thera...
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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.