MedPage Today11d
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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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 ...
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MaxCyte: Building the Future of Cell and Gene Therapy Innovation
Gene editing tools like CRISPR/Cas-9 molecular scissors are delivered ... Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused ...
The Scientist5y
Early Results Are Positive for Experimental CRISPR Therapies
The therapy, known as CTX001, is applied to blood stem cells removed from the patient to cleave the BCL11A gene that represses the production of fetal hemoglobin. These cells are then reinfused to ...
Nasdaq26d
MaxCyte: Building the Future of Cell and Gene Therapy Innovation
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 ...