The effectiveness of CRISPR hinges on delivery efficiency and specificity. Delivery vehicles include:
Viral vectors (e.g., AAV) that can efficiently transport CRISPR components into cells,
Non‑viral systems like lipid nanoparticles, which reduce immunogenicity and improve safety profiles. PubMed
Off‑target effects—unintended edits in the genome—remain a primary safety concern. Computational tools and high‑fidelity Cas variants are continuously developed to lower these risks and improve clinical safety.