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Abstract
Prime editing may correct 89% of human pathogenic mutations associated with genetic diseases.
- Prime editing is a CRISPR-Cas9-derived technology that enables precise base conversions without causing double-strand breaks.
- There are significant challenges in translating prime editing from preclinical models to clinical applications, particularly with in vivo delivery systems.
- Therapeutic outcomes of in vivo prime editing have been observed in various genetic diseases, including retinal, metabolic, and hematologic disorders.
- Barriers to clinical translation include the immunogenicity of viral vectors and off-target risks in nondividing cells.
- Large-scale production bottlenecks exist that may hinder the implementation of in vivo prime editing therapies.
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