Introduction The emergence of CRISPR-Cas9 technology has transformed genome engineering by enabling precise gene editing. Guided by single-guide RNAs (gRNAs), the Cas9 nuclease induces site-specific […]
Category: therapy
AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids
Dear Editor, Genome editing holds potential to cure human diseases. The first CRISPR/Cas9 gene therapy, Casgevy, may offer a definitive treatment for hemoglobinopathies through autologous […]