Full-speed mammalian genetics: in vivo target validation in the drug discovery process.
The completion of the Human Genome Project has signaled the beginning of the post-genome era, with a corresponding shift in focus from the sequencing and identification of genes to the exploration of gene function. A rate-limiting step in deriving value from this gene sequence information is determining the potential pharmaceutical applications of genes and their encoded proteins. This validation step is crucial for focusing efforts and resources on only the most promising targets. Strategies using reverse mouse genetics provide excellent methods for validating potential targets and therapeutic proteins in vivo in a mammalian model system.
Abuin
Alejandro
A
Lexicon Genetics Incorporated, 4000 Research Forest Drive, The Woodlands, TX 77381, USA.
Holt
Kathleen H
KH
Platt
Kenneth A
KA
Sands
Arthur T
AT
Zambrowicz
Brian P
BP
D000818:Animals; D002616:Chemical Engineering / Q000379:methods / Q000592:standards; D015195:Drug Design; D004353:Drug Evaluation, Preclinical / Q000592:standards*; D005260:Female; D005544:Forecasting; D018390:Gene Targeting / Q000379:methods / Q000592:standards* / Q000639:trends; D005818:Genetic Engineering / Q000379:methods* / Q000592:standards*; D005820:Genetic Testing / Q000592:standards* / Q000639:trends; D015894:Genome, Human; D006801:Humans; D008297:Male; D051379:Mice; D008822:Mice, Transgenic; D023421:Models, Animal; D016296:Mutagenesis / Q000235:genetics; D010641:Phenotype