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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...

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Related Experiment Video

Updated: Jul 5, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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Gene delivery to the liver.

K Kozarsky1

  • 1SmithKline Beecham Pharmaceuticals, Inc., King of Prussia, Pennsylvania, USA.

Current Protocols in Human Genetics
|April 23, 2008
PubMed
Summary

Viral gene transfer effectively targets liver cells in animal models, showing promise for treating metabolic disorders. Optimized protocols for adenoviral vector delivery in rodents are detailed for research applications.

Area of Science:

  • Hepatology
  • Gene Therapy
  • Viral Vector Technology

Background:

  • Viral gene transfer to the liver is a promising therapeutic strategy for metabolic disorders.
  • Adenoviral vectors are effective tools for in vivo gene delivery in rodent models.

Purpose of the Study:

  • To describe protocols for optimized in vivo hepatic gene transfer using adenoviral vectors.
  • To highlight the utility of rodent models for assessing vector design and transgene effects.

Main Methods:

  • Adenoviral vector administration via tail vein injection in rodents.
  • Assessment of hepatocyte transduction efficiency in vivo.

Main Results:

  • A single tail vein injection of adenoviral vectors efficiently transduces hepatocytes in vivo in rodents.

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  • This method provides a robust model for evaluating gene transfer efficacy and vector performance.

    • Conclusions:

    • Optimized in vivo hepatic gene transfer protocols using adenoviral vectors are established.
    • Rodent models are valuable for advancing gene therapy research for metabolic diseases.