Hosted on MSN1mon
Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
In new work just published in Science, Orkin and his team at Dana-Farber/Boston Children's Cancer and Blood Disorder ... It does so by suppressing the gene BCL11A, which normally shuts down ...
Nasdaq22d
MaxCyte: Building the Future of Cell and Gene Therapy Innovation
For cell therapy, they help engineer immune cells like CAR-T cells to fight cancer. Gene editing tools ... Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal ...