Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Validating a Human Cell Model of Null Galactosylceramidase (GALC) Enzyme Activity That Recapitulates Krabbe Disease.

Journal of inherited metabolic disease·2026
Same author

Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease.

International journal of molecular sciences·2026
Same author

Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice.

Acta neuropathologica communications·2025
Same author

Neuronal ceroid lipofuscinosis: underlying mechanisms and emerging therapeutic targets.

Nature reviews. Neurology·2025
Same author

William S. Sly (1932-2025): A compassionate pioneer in biochemical genetics.

Proceedings of the National Academy of Sciences of the United States of America·2025
Same author

GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice.

JCI insight·2025

Related Experiment Video

Updated: May 28, 2026

Efficient Gene Knockdown in the Liver via Intrasplenic Injection of Adeno-Associated Virus Serotype 8 (AAV8)-Delivered Small Hairpin RNA
04:29

Efficient Gene Knockdown in the Liver via Intrasplenic Injection of Adeno-Associated Virus Serotype 8 (AAV8)-Delivered Small Hairpin RNA

Published on: November 1, 2024

AAV-mediated liver-directed gene therapy.

Mark S Sands1

  • 1Washington University School of Medicine, St. Louis, MO, USA. msands@dom.wustl.edu

Methods in Molecular Biology (Clifton, N.J.)
|October 29, 2011
PubMed
Summary

Adeno-associated viral (AAV) vectors, particularly AAV8, show promise for liver gene therapy by efficiently targeting hepatocytes. This approach offers a potential strategy for treating inherited liver diseases with improved gene expression and reduced immune responses.

Area of Science:

  • Hepatology and Gene Therapy
  • Viral Vector Technology

Background:

  • The liver's central role in metabolism and its susceptibility to inherited diseases necessitate effective gene therapy strategies.
  • Gene transfer targeting hepatocytes faces challenges including efficient cell targeting, vector genome stability, and sustained high-level expression.

Purpose of the Study:

  • To evaluate the efficacy of adeno-associated viral (AAV) vectors, specifically different serotypes, for liver-directed gene therapy.
  • To compare the hepatocyte transduction efficiency of AAV2, AAV8, and AAV9 serotypes for potential therapeutic applications.

Main Methods:

  • Utilized adeno-associated viral (AAV) gene transfer vectors, focusing on serotypes AAV2, AAV8, and AAV9.
  • Administered AAV vectors via intraportal vein, intravenous, and direct intraparenchymal injections in mouse models.

More Related Videos

High-Efficiency Transduction of Liver Cancer Cells by Recombinant Adeno-Associated Virus Serotype 3 Vectors
19:02

High-Efficiency Transduction of Liver Cancer Cells by Recombinant Adeno-Associated Virus Serotype 3 Vectors

Published on: March 22, 2011

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
09:54

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

Published on: November 4, 2018

Related Experiment Videos

Last Updated: May 28, 2026

Efficient Gene Knockdown in the Liver via Intrasplenic Injection of Adeno-Associated Virus Serotype 8 (AAV8)-Delivered Small Hairpin RNA
04:29

Efficient Gene Knockdown in the Liver via Intrasplenic Injection of Adeno-Associated Virus Serotype 8 (AAV8)-Delivered Small Hairpin RNA

Published on: November 1, 2024

High-Efficiency Transduction of Liver Cancer Cells by Recombinant Adeno-Associated Virus Serotype 3 Vectors
19:02

High-Efficiency Transduction of Liver Cancer Cells by Recombinant Adeno-Associated Virus Serotype 3 Vectors

Published on: March 22, 2011

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
09:54

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

Published on: November 4, 2018

  • Employed liver-specific promoters in conjunction with AAV8 capsid proteins to enhance targeting specificity.

    • Main Results:

    • AAV8 and AAV9 demonstrated higher affinities for hepatocytes compared to AAV2.
    • AAV8 achieved significantly higher hepatocyte transduction rates (3-4 fold) and genome delivery per cell than AAV2.
    • Up to 90-95% hepatocyte transduction was observed with AAV8 following intraportal injection, with comparable results via intravenous administration.

    Conclusions:

    • AAV8 and AAV9 represent superior serotypes for liver-directed gene therapy due to enhanced hepatocyte tropism and transduction efficiency.
    • Combining AAV8 serotype with liver-specific promoters offers a robust strategy for targeted gene delivery to hepatocytes, minimizing immune reactions.
    • This approach holds significant potential for treating inherited liver disorders and advancing fundamental research in liver biology and disease.