| 摘要 |
A method of validating potential drug targets is provided. A potential drug target (or an inhibitor thereof) is introduced into animal lines such that the expression of the target is spatially or temporarily restricted. The animal lines are transgenic for a "key gene" which encodes a "key protein" that activates or inhibits expression of the potential target. Key protein expression is regulated by regulatory sequences from a "characterizing gene," whose expression is spatially or temporarily restricted. For target validation, a "modulating construct" containing a nucleotide sequence encoding the target, or a product that modulates the expression of the target, is introduced into a transgenic cell line with a desired key gene expression pattern. Expression of the target, or modulator thereof, is regulated by the presence of the key protein, so that only cells expressing the key protein can activate or inhibit expression of the drug target.
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