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Using siRNA technique to generate transgenic animals with spatiotemporal and conditional gene knockdown.

Based on the RNAi technique, we have developed a new approach that generates transgenic animals capable of mimicking human genetic diseases. The new system is a combination of siRNA with Cre-loxP and tetracycline-on. It has the characteristics of being stable, inheritable, and inducible, with the siRNA able to be transcribed tissue specifically. To support the ability of this new method to generate a model for a disease, we created an ABCA1-deficient mouse line that mimics Tangier disease under controlled conditions. Thus, it should now be possible to rapidly establish human genetic diseases as a whole animal model without the use of embryonic stem cell and gene targeting. This system also provides a tool for pathological and pharmacological studies of aspects peculiar to particular human genetic diseases.

Pubmed ID: 15509524 RIS Download

Mesh terms: ATP Binding Cassette Transporter 1 | ATP-Binding Cassette Transporters | Animals | Cell Line, Tumor | Cholesterol | Disease Models, Animal | Doxycycline | Genetic Techniques | Genetic Vectors | Humans | Mice | Mice, Inbred ICR | Mice, Knockout | Mice, Transgenic | Models, Genetic | Phenotype | Plasmids | RNA Interference | RNA, Small Interfering | Tangier Disease | Time Factors | Tissue Distribution | Transfection

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