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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: Jul 20, 2026

Loss-of-Function Approach in the Embryonic Chick Retina by Using Tol2 Transposon-Mediated Transgenic Expression of Artificial microRNAs

Loss-of-Function Approach in the Embryonic Chick Retina by Using Tol2 Transposon-Mediated Transgenic Expression of Artificial microRNAs

Published on: May 18, 2022

Transgene-like animal models using intronic microRNAs.

Shi-Lung Lin¹, Shin-Ju E Chang, Shao-Yao Ying

¹Department of Cell and Neurobiology, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.

Methods in Molecular Biology (Clifton, N.J.)

|September 8, 2006

Summary

Researchers developed a novel transgenic animal model strategy using intronic microRNAs (miRNAs) to silence specific genes. This breakthrough enables crucial in vivo research for microRNA function and therapeutic development.

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Last Updated: Jul 20, 2026

Loss-of-Function Approach in the Embryonic Chick Retina by Using Tol2 Transposon-Mediated Transgenic Expression of Artificial microRNAs

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Published on: May 18, 2022

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Published on: June 15, 2017

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Published on: October 7, 2018

Area of Science:

Molecular Biology
Genetics
Biotechnology

Background:

Transgenic animal models are essential for in vivo research, drug testing, and disease modeling.
MicroRNAs (miRNAs) are regulatory RNAs with therapeutic potential, but lack dedicated in vivo models.
Existing research highlights miRNA origins and functions across diverse eukaryotic species.

Purpose of the Study:

To develop a state-of-the-art transgenic strategy for in vivo microRNA research.
To create functional transgenic animal models for studying gene silencing via intronic miRNAs.
To enable in vivo evaluation of miRNA-associated target genes and therapeutic development.

Main Methods:

Developed a transgenic strategy utilizing intronic microRNAs (miRNAs) for gene silencing.
Inserted hairpin-like pre-miRNA structures into gene introns for co-expression and processing.
Employed retroviral transfection for tissue-specific expression and selection of stable transgenic lines.

Main Results:

Successfully generated mature miRNAs transcribed by RNA polymerase II and co-expressed with host genes.
Demonstrated natural RNA splicing and processing mechanisms for miRNA excision.
Created the first transgene-like animal models using an intronic miRNA-expressing system.

Conclusions:

The intronic miRNA-expressing system provides a novel and effective method for generating transgenic animal models.
This system is valuable for both fundamental microRNA research and in vivo drug evaluation.
The developed models facilitate the study of miRNA-associated target genes and therapeutic applications.