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Am J Pathol. 2004 Nov;165(5):1535-41.

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

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  • 1National Health Research Institutes, 3F, 109, Min-Chuan East Road, Sec 6, Taipei 114, Taiwan, ROC.

Abstract

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.

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