Application of CRISPR-Cas9-Based Gene Editing Technology in Inherited Liver Diseases

Area of Science:

• Biotechnology
• Genetics
• Hepatology

Background:

• Inherited liver diseases stem from monogenic mutations, with limited curative treatments.
• Liver transplantation is an option but has significant challenges like donor shortage and lifelong immunosuppression.
• CRISPR-Cas9 gene editing technology presents a promising therapeutic avenue.

Purpose of the Study:

• To review the principles and advantages of CRISPR-Cas9 and its variants (base and prime editing).
• To summarize current applications of gene editing in treating hereditary liver diseases.
• To discuss the prospects and challenges for clinical translation of these gene editing therapies.

Main Methods:

• Systematic review of CRISPR-Cas9 technology and its derived tools.
• Analysis of recent research on gene editing applications for inherited liver diseases.
• Discussion of clinical translation prospects and hurdles.

Main Results:

• CRISPR-Cas9, base editing, and prime editing offer precise gene modification capabilities.
• These technologies have shown potential in preclinical models for various hereditary liver conditions.
• Significant progress has been made in applying gene editing to liver disease targets.

Conclusions:

• Gene editing technologies hold transformative potential for treating inherited liver diseases.
• Further research and development are crucial to overcome challenges for clinical translation.
• This review provides a reference for future research in gene editing for liver diseases.