Off-target effects, where CRISPR/Cas9 may unintentionally edit other parts of the genome, could have serious consequences, including the potential for oncogenesis.29Advances in gRNA design and high-fidelity Cas9 variants have reduced these risks, but rigorous testing in clinical trials is essential to confirm the safety and efficacy of these therapies.17Addressing these delivery and safety challenges is crucial for the successful translation of CRISPR/Cas9 therapies from bench to bedside, paving the way for clinical trials specifically targeting the dystrophin gene in DMD patients. This evidence concerns the gene DMD and Duchenne muscular dystrophy.