[Research progress of CRISPR-Cas9 system for gene therapy]

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• The clustered regulatory interspaced short palindromic repeat-Cas9 (CRISPR-Cas9) system originates from prokaryotic immune mechanisms.
• It naturally targets and eliminates foreign DNA sequences from viruses and plasmids.

Purpose of the Study:

• To review the advancements in CRISPR-Cas9 technology.
• To explore its latest applications in gene therapy for human diseases.

Main Methods:

• Review of existing scientific literature on CRISPR-Cas9.
• Analysis of CRISPR-Cas9 applications in human disease gene therapy.

Main Results:

• CRISPR-Cas9 is a highly efficient, economical, and promising gene editing tool.
• It surpasses older technologies like ES targeting, ZFN, and TALEN in genome manipulation.

Conclusions:

• CRISPR-Cas9 represents a significant breakthrough in gene editing technology.
• Its application in human gene therapy holds immense potential for treating diseases.