Prime editing: A gene precision editing tool from inception to present.

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• Genetic mutations, particularly single-nucleotide mutations, are primary drivers of numerous diseases.
• Gene editing technologies have emerged as powerful tools for correcting mutations and treating genetic disorders.
• CRISPR/Cas9, while effective, carries risks of off-target mutations due to DNA double-strand breaks (DSB).

Purpose of the Study:

• To review the principles, development, and applications of prime editing (PE) technology.
• To highlight PE's advantages over other gene editing methods, especially in mitigating risks associated with DNA DSBs.
• To explore the potential of PE in treating diseases caused by genetic mutations.

Main Methods:

• Discussion of the underlying mechanisms of prime editing.
• Analysis of the evolution from CRISPR/Cas9 to base editing (BE) and prime editing (PE).
• Review of current and potential applications of PE in various genetic disease models.

Main Results:

• Prime editing enables precise gene correction without inducing DNA double-strand breaks.
• Base editing and prime editing significantly reduce the risks of unintended mutations compared to CRISPR/Cas9.
• PE technology demonstrates broad applicability for correcting a wide range of genetic mutations.

Conclusions:

• Prime editing represents a significant advancement in gene editing technology, offering enhanced precision and safety.
• The elimination of DNA DSB hazards makes PE a promising tool for clinical gene therapy.
• Further development and application of PE hold substantial potential for treating diverse genetic diseases.