Discover genetics and evolution2026ReviewNon-viral Gene Delivery
1. Navigating off-target effects in CRISPR-based genome editing for safer gene therapies
Bhardwaj A
Off-target activity remains a significant challenge in the clinical development of CRISPR-based therapies. As programmable nucleases advance from experimental tools toward approved medicines, the ability to predict, detect, and mitigate unintended genomic editing events has acquired direct translational importance.
- Base editing
- CRISPR-Cas systems
- Epigenome editing
- Gene therapy
- Genome editing
- Guide RNA design
- High-fidelity Cas9
- Off-target effects
- Prime editing
- Structural variation
