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Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.2K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Liver Regeneration01:24

Liver Regeneration

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The liver is an important organ in vertebrates that plays an essential role in metabolism. It is also responsible for storing and redistributing nutrients such as carbohydrates, fats, and vitamins in the body. Additionally, the liver releases bile salts which are critical for digesting food and eliminating toxic metabolites from the body.
Cells of Liver
The liver comprises four major types of cells— hepatocytes, stellate, Kupffer, and sinusoidal endothelial cells. The hepatocytes are...
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Cell Specific Gene Expression01:58

Cell Specific Gene Expression

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Multicellular organisms contain a variety of structurally and functionally distinct cell types, but the DNA in all the cells originated from the same parent cells. The differences in the cells can be attributed to the differential gene expression. Liver cells, whose functions include detoxification of blood, production of bile to metabolize fats, and synthesis of proteins essential for metabolism, must express a specific set of genes to perform their functions. Gene expression also varies with...
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Updated: Jun 4, 2025

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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Gene Therapy for Inherited Liver Disease: To Add or to Edit.

Yue Chen1, Niek P van Til2,3, Piter J Bosma1

  • 1Amsterdam University Medical Center, Tytgat Institute for Liver and Intestinal Research, AG&M, University of Amsterdam, Meibergdreef 69-71, 1105 BK Amsterdam, The Netherlands.

International Journal of Molecular Sciences
|December 17, 2024
PubMed
Summary

Gene therapy offers a less invasive alternative to liver transplantation for inherited liver disorders. This review explores gene supplementation, editing, and repair strategies for treating these severe genetic conditions.

Keywords:
gene augmentationgenome editinginherited liver diseases

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Area of Science:

  • Hepatology
  • Genetics
  • Molecular Biology

Background:

  • Inherited severe liver disorders necessitate lifelong treatment, with liver transplantation historically being the only cure, requiring immunosuppression.
  • Liver-directed gene therapy is now approved for hemophilia A and B, indicating its potential for other genetic liver diseases.
  • Alternative gene therapy approaches are needed for liver diseases involving toxic gain-of-function or intrinsic hepatocyte damage.

Purpose of the Study:

  • To provide an overview of current gene therapy strategies for inherited severe liver disorders.
  • To discuss the advantages and limitations of gene therapy applications based on disease mechanisms.

Main Methods:

  • Review of preclinical and clinical studies on gene supplementation, gene editing, and gene repair for inherited liver diseases.
  • Analysis of disease mechanisms to evaluate suitability of different gene therapy approaches.

Main Results:

  • Gene supplementation is a viable strategy for loss-of-function mutations, as demonstrated by hemophilia treatments.
  • Gene editing and integrating vectors offer potential for permanent genomic modification in toxic gain-of-function or intrinsic damage models.
  • The choice of gene therapy strategy depends critically on the specific genetic defect and disease mechanism.

Conclusions:

  • Liver-directed gene therapy presents a promising, less invasive treatment paradigm for inherited severe liver disorders.
  • Gene supplementation, editing, and repair technologies are advancing, with tailored applications emerging for diverse genetic liver conditions.
  • Further research and clinical translation are essential to optimize gene therapy for a broader range of inherited liver diseases.