Teams at the University of Washington School of Pharmacy and Fred Hutch will model the clinical and economic burden of sickle cell disease and the potential benefits of emerging therapies
A new Science Translational Medicine paper is first to report how editing a portion of stem cells with CRISPR/Cas9 is sufficient for long-term reactivation of therapeutic hemoglobin
A proof-of-principle study published in Nature Materials shows that gold nanoparticles loaded with CRISPR and other gene-editing tools safely and effectively edited blood stem cells. (Photos, diagram available for media use.)