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Related Concept Videos

In-vitro Mutagenesis01:16

In-vitro Mutagenesis

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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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Related Experiment Video

Updated: Jan 12, 2026

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

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In-Depth Optimization and Systematic Evaluation of a Recombinant AAV-Based Hepatic-Targeted Gene Knockdown Murine

Ying Zhu1,2,3, Guohua You4, Yong Jiang5

  • 1Department of Hepatobiliary and Pancreatic Surgery, the First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, China.

FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology
|October 30, 2025
PubMed
Summary

A new, rapid, and cost-effective recombinant adeno-associated virus (rAAV) tool enables liver-specific gene knockdown in mice. This optimized model accelerates preclinical liver research and therapeutic development.

Keywords:
Kupffer cellsliver sinusoidal endothelial cellsliver‐specific knockdownmouse modelrecombinant adeno‐associated virus

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Area of Science:

  • * Molecular Biology
  • * Gene Therapy
  • * Preclinical Research

Background:

  • * Precise gene-edited animal models are crucial for liver research but are often expensive and time-consuming.
  • * Developing rapid and economical models is essential for advancing preclinical studies.

Purpose of the Study:

  • * To establish a liver-specific gene knockdown mouse model using a recombinant adeno-associated virus (rAAV) tool.
  • * To optimize delivery methods and evaluate knockdown efficiency for hepatic ischemia/reperfusion (IR) injury-related genes (Btg2, Flrt3, Klf4).

Main Methods:

  • * Engineered rAAV vectors with a liver-specific promoter (TBG669), Firefly luciferase (Fluc) for tracking, and miR30 regulatory elements.
  • * Optimized delivery routes (tail vein vs. dorsal penile vein) and doses (2.5 × 10^11 GC vs. 5.0 × 10^11 GC).
  • * Assessed safety and knockdown via histopathology, serum biochemistry, qRT-PCR, and western blot in bulk and single-cell liver tissues.

Main Results:

  • * Optimized rAAV system achieved 100% hepatic infection with minimal off-target effects.
  • * Dorsal penile vein injection proved efficient and simplified delivery.
  • * Full-dose administration enhanced bioluminescence without adverse effects; Btg2 and Flrt3 showed >50% knockdown, Klf4 moderate suppression.
  • * Effective knockdown observed in Kupffer cells, hepatocytes, and liver sinusoidal endothelial cells (LSECs).

Conclusions:

  • * Established a time- and cost-efficient rAAV-based murine model for hepatic gene knockdown.
  • * This model facilitates precise investigation of liver disease mechanisms.
  • * Enables accelerated development of novel liver therapeutics.