Full text is available at the source.
Abstract
Clinical trials have reported up to 93% protein knockdown after a single dose of nonviral lipid nanoparticles for hepatic targets.
- Therapeutic genome editing has progressed with various programmable nucleases, including CRISPR-based systems.
- Clinical translation of these technologies faces challenges in safe, efficient, and tissue-specific delivery.
- Viral vectors like adeno-associated viruses have shown durable editing in specific organs but have limitations in cargo capacity and immunogenicity.
- Emerging delivery modalities include virus-mimicking nanosystems and cell-derived extracellular vesicles, enhancing transient expression and programmable targeting.
- Advances in high-throughput screening and machine learning are aiding in the optimization of delivery vectors.
Simplified