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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
Casgevy works by enabling people to produce a fetal form of hemoglobin that doesn't cause sickling. It does so by suppressing the gene BCL11A, which normally shuts down fetal hemoglobin production ...
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Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
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MaxCyte: Building the Future of Cell and Gene Therapy Innovation
Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into a patient's bone marrow to start producing red blood cells with high ...
Nasdaq22d
MaxCyte: Building the Future of Cell and Gene Therapy Innovation
Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into a patient's bone marrow to start producing red blood cells with ...