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Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.7K
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

3.3K
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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Liver Physiology01:30

Liver Physiology

921
The liver, an essential organ in the human body, performs over 200 vital functions that can be broadly categorized into metabolic, hematological, endocrine regulation, and bile production.
Metabolic Regulation:
The liver is the central organ involved in regulating blood composition. It stabilizes blood glucose levels, maintaining them within the range of  70–110 mg/dL. When these levels drop, the liver breaks down glycogen reserves and releases glucose into the bloodstream. It can...
921
Diseases of the Liver and Gallbladder01:26

Diseases of the Liver and Gallbladder

902
Liver and gallbladder diseases are a significant health concern, with prominent conditions including cirrhosis, hepatitis, non-alcoholic fatty liver disease (NAFLD), and gallstones. Jaundice is a common manifestation of liver and biliary disease.
Cirrhosis is characterized by the scarring of hepatic lobules in the liver, which are replaced by fibrous tissue, affecting the liver's normal functioning. NAFLD, on the other hand, is caused by an excessive build-up of fat in the liver, not...
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Cell Specific Gene Expression01:58

Cell Specific Gene Expression

13.7K
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...
13.7K

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Alternative AAV gene therapy for hemophilia A using expression of Bi8, a novel single-chain FVIII-mimetic antibody.

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Related Experiment Video

Updated: Aug 29, 2025

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

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Liver Gene Therapy.

Amit C Nathwani1,2,3, Jenny McIntosh1,2, Rose Sheridan3

  • 1Department of Haematology, UCL-Cancer Institute, London, United Kingdom.

Human Gene Therapy
|September 9, 2022
PubMed
Summary

Gene therapy offers potential cures for liver disorders. Recent advancements show promising clinical efficacy and safety, marking a new era for molecular medicine treatments.

Keywords:
adeno-associated virus (AAV)gene therapylivermonogeneticreview

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Area of Science:

  • Hepatology
  • Molecular Medicine
  • Gene Therapy

Background:

  • The liver's central role in metabolism and essential biological processes makes it a prime target for gene therapy.
  • Numerous inherited metabolic disorders of hepatic origin currently lack cures, with liver transplantation often being the only option.
  • Significant progress in liver-targeted gene therapy has been achieved in the last decade, moving beyond previous failures.

Purpose of the Study:

  • To review recent clinical advances in liver-targeted gene therapy.
  • To explore the challenges hindering the widespread adoption of this therapeutic approach.

Main Methods:

  • Review of clinical trial data and regulatory approvals for liver-targeted gene therapies.
  • Analysis of scientific literature on the efficacy and safety of gene therapy for hepatic disorders.

Main Results:

  • Valoctocogene roxaparvovec (Roctavian) for hemophilia A and Etranacogene dezaparvovec (AMT-061) for hemophilia B represent major breakthroughs, achieving regulatory approval or nearing it.
  • Several other liver-targeted gene therapy candidates are in advanced development stages.
  • Demonstrated clinical efficacy and safety in patients with monogenetic liver disorders.

Conclusions:

  • Liver-targeted gene therapy is emerging as a powerful, potentially curative treatment modality for various hepatic disorders.
  • Overcoming existing challenges is crucial for the widespread clinical adoption of these innovative therapies.
  • A new era of curative molecular medicine is dawning, driven by advancements in gene therapy.