Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders

Area of Science:

• Hepatology
• Genetics
• Molecular Biology

Background:

• Most monogenic liver diseases are autosomal recessive, with limited treatment options beyond liver transplantation (LT).
• LT is restricted by donor availability and significant post-transplant complications, including the need for lifelong immunosuppression.
• Innovative cellular and gene-based therapies are being developed to address these limitations.

Purpose of the Study:

• To review common monogenic liver diseases.
• To explore genetic engineering approaches as potential therapeutic modalities.
• To highlight advancements in gene therapy for hereditary liver disorders.

Main Methods:

• Review of current literature on monogenic liver diseases.
• Analysis of gene therapy strategies, including viral and non-viral vectors.
• Evaluation of gene-editing technologies such as CRISPR-Cas9.

Main Results:

• Gene therapy, particularly with gene-editing tools, shows promise for treating monogenic liver diseases.
• The liver's unique characteristics make it a suitable target for in vivo and ex vivo gene transfer.
• Despite progress, challenges remain in developing validated gene correction approaches.

Conclusions:

• Gene therapy represents a viable and evolving therapeutic strategy for monogenic liver diseases.
• Further refinement of gene therapy protocols is crucial for clinical application.
• Genetic engineering holds significant potential for managing hereditary liver conditions.