CRISPR Center Launches with $20M to Develop Personalized Therapies

Recent advances in CRISPR gene-editing technology are rapidly transforming the treatment landscape for rare genetic diseases, particularly in children. The Center for Pediatric CRISPR Cures at UCSF, backed by $20 million from the Chan Zuckerberg Initiative and led by Nobel laureate Jennifer Doudna, is launching clinical trials for personalized CRISPR therapies targeting rare pediatric genetic disorders, inspired by the successful treatment of infant KJ Muldoon with a customized base-editing therapy. Meanwhile, research at the University of Chicago has developed highly precise cytosine base editors that minimize off-target effects by evolving nucleotide specificity, promising more accurate therapeutic genome editing. Additionally, a new software tool called CRISPRware, created by a UC Santa Cruz Ph.D. student, enhances the design of guide RNAs for genome editing, particularly in unannotated genomic regions, broadening the scope for precision gene therapies. These innovations collectively highlight the accelerating pace and increasing precision of CRISPR-based treatments, aiming to make gene editing a widely accessible option for previously untreatable genetic disorders.