[Recent advances and applications of base editing systems]

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• CRISPR systems offer precise genomic DNA editing but are limited by reliance on inefficient homology-directed repair.
• Base editing, a novel genome editing technology, has been developed using the CRISPR/Cas9 system.
• Existing methods often result in DNA double-stranded breaks, limiting their efficiency and applicability.

Purpose of the Study:

• To review the development, technical advantages, applications, challenges, and future perspectives of base editing.
• To provide a comprehensive understanding of base editing for researchers.
• To highlight base editing as a powerful tool for basic and applied research.

Main Methods:

• Development of base editors by fusing catalytically disabled CRISPR/Cas9 nucleases with base-specific deaminases.
• Utilizing cytosine base editors (C>T or G>A transitions) and adenine base editors (A>G or T>C transitions).
• Performing base conversions without inducing DNA double-stranded breaks.

Main Results:

• Two types of base editors (cytosine and adenine) have been successfully developed.
• Base editing allows for precise transition mutations (C>T, G>A, A>G, T>C) without DNA double-stranded breaks.
• The technique has demonstrated broad applicability in various research fields.

Conclusions:

• Base editing represents a significant advancement in genome editing technology.
• Its ability to precisely modify DNA without double-stranded breaks offers advantages over traditional methods.
• Base editing is a versatile tool with wide-ranging applications in gene therapy, animal models, breeding, and functional genomics.