The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
Hosted on MSN1mon
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 ...
Hosted on MSN22d
MaxCyte: Building the Future of Cell and Gene Therapy InnovationGene 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 ...
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 ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback