CRISPR Rewrites the Future of Medicine

摘要

This themed issue on “CRISPR's Path to the Clinic” illustrates how quickly genome editing and CRISPR technology development have turned into early-stage clinical endeavors. Less than a decade after CRISPR-based programmable genome-editing technologies were developed, the reality of Cas-fueled gene therapies in the clinic is apparent. Indeed, encouraging Phase I results for sickle cell disease (SCD), β-thalassemia, and transthyretin amyloidosis have set the stage for a series of trials to recruit and dose patients throughout 2022. CRISPR is literally writing the future of medicine in patients' genomes.