CRISPR-Cas9 System as a Versatile Tool for Genome Engineering in Human Cells

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• Genome editing technologies are crucial for biological research and therapeutic development.
• RNA-guided Cas9 nucleases, derived from CRISPR-Cas systems, provide unprecedented accuracy in modifying genomes.
• Understanding the CRISPR-Cas system is vital for advancing genetic engineering.

Approach:

• This review discusses the molecular mechanisms underlying the type II CRISPR/Cas system.
• It examines the applications and influencing factors of CRISPR-Cas9 in human cells.
• Recent advancements and future prospects are explored.

Key Points:

• CRISPR-Cas9 enables targeted genome manipulation in eukaryotic cells via a 20-nt guide RNA.
• The type II CRISPR/Cas system's molecular basis is detailed.
• Applications range from basic research to clinical implementation.

Conclusions:

• CRISPR-Cas9 technology has revolutionized genome engineering.
• Its diverse applications hold significant promise for both scientific discovery and clinical medicine.
• Further research into its molecular basis and applications will expand its utility.