In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long ...
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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug"The editing therapy is a wonderful achievement ... It does so by suppressing the gene BCL11A, which normally shuts down fetal hemoglobin production after birth. This lets fetal hemoglobin ...
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
Gene editing tools like CRISPR/Cas-9 molecular scissors ... Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into ...
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