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Abstract
Recent clinical trials have demonstrated the feasibility and safety of gene editing technologies like CRISPR-Cas9 in various disease areas.
- Gene editing may address a wide range of genetic and acquired diseases.
- Advanced techniques such as zinc-finger nucleases and base editors are being explored in clinical applications.
- Efficient delivery systems, including lipid nanoparticles and viral vectors, are crucial for successful gene editing.
- Early-phase trials have shown durable gene modification in patient populations.
- Challenges remain regarding delivery specificity and minimizing off-target effects.
- Ongoing trials aim to enhance delivery technologies and expand therapeutic applications.
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