Harvard Medical School16h
Creating the World’s First CRISPR Medicine, for Sickle Cell Disease
In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long ...
MedPage Today13d
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 ...
Hosted on MSN2mon
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 MSN28d
MaxCyte: Building the Future of Cell and Gene Therapy Innovation
which is critical for the effectiveness of the therapy. 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 ...