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Related Experiment Video

Updated: Jun 6, 2026

Consistent Delivery of Adeno-Associated Virus via Lateral Tail-Vein Injection in Adult Mice
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Consistent Delivery of Adeno-Associated Virus via Lateral Tail-Vein Injection in Adult Mice

Published on: August 23, 2024

Assessing the potential for AAV vector genotoxicity in a murine model.

Hojun Li1, Nirav Malani, Shari R Hamilton

  • 1Department of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.

Blood
|November 26, 2010
PubMed
Summary

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Adeno-associated virus (AAV) gene therapy shows safety in mice. Studies found no increased tumor formation or oncogene activation from AAV vector DNA integration, supporting its therapeutic potential.

Area of Science:

  • Molecular Biology
  • Genetics
  • Translational Medicine

Background:

  • Adeno-associated virus (AAV) vectors are promising for gene therapy.
  • Concerns exist regarding AAV vector DNA integration and potential tumor formation.

Purpose of the Study:

  • To investigate the safety of high-dose liver-directed AAV gene transfer in mice.
  • To determine if AAV vector DNA integration promotes hepatocellular carcinoma.

Main Methods:

  • High-dose liver-directed AAV gene transfer in adult mice (80 AAV-injected, 52 controls).
  • 18-month follow-up for tumor development and hepatocellular carcinoma rates.
  • Development of a high-throughput method to identify and analyze AAV vector integration sites.
  • Analysis of gene expression near integration sites.

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Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors
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Last Updated: Jun 6, 2026

Consistent Delivery of Adeno-Associated Virus via Lateral Tail-Vein Injection in Adult Mice
05:03

Consistent Delivery of Adeno-Associated Virus via Lateral Tail-Vein Injection in Adult Mice

Published on: August 23, 2024

In Vitro Establishment of a Genetically Engineered Murine Head and Neck Cancer Cell Line using an Adeno-Associated Virus-Cas9 System
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In Vitro Establishment of a Genetically Engineered Murine Head and Neck Cancer Cell Line using an Adeno-Associated Virus-Cas9 System

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Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors
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Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors

Published on: January 29, 2019

Main Results:

  • No significant increase in hepatocellular carcinoma in AAV-injected mice compared to controls.
  • Vector DNA levels in tumors were not significantly different from normal tissue.
  • Identified 1029 AAV integration sites, with similar patterns in tumor and normal tissues.
  • No significant changes in gene expression near integration sites; no oncogene activation observed.

Conclusions:

  • AAV vectors did not demonstrate increased tumor formation in this mouse model.
  • No evidence suggests AAV vector DNA integration causes insertional activation of oncogenes.
  • Findings support the safety of AAV vectors for gene therapy applications.