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Abstract
CRISPR-Cas systems are increasingly recognized for their potential in targeted genome and transcriptome engineering.
- Recent advances include base editing, prime editing, and epigenome modulation, expanding therapeutic options beyond traditional DNA cutting.
- Early clinical studies have shown sustained therapeutic benefits for certain monogenic disorders using both ex vivo and in vivo editing methods.
- Challenges persist, including off-target effects, difficulties in delivering the editing tools, immune reactions to Cas proteins, and variability in editing outcomes.
- There are uncertainties about the long-term safety of CRISPR technologies in clinical applications.
- This review highlights both the technological progress and the limitations that need to be addressed for effective CRISPR-based therapies.
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