Validation of GABA(A) receptor subtypes as potential drug targets by using genetically modified mice.

摘要: A key issue for drug discovery in the post genomic era is target validation. This particularly important when considering CNS, where currently majority of targets are neurotransmitter receptors that known to exist as multi-gene families. The GABAergic system, which major inhibitory system no exception respect. GABA(A) receptors, site action a number clinically used drugs such benzodiazepines and barbiturates, large gene family. Existing mediating their effects through receptor generally non-selective, i.e. will act at several subtypes Thus, if we both refine existing therapeutic approaches, develop novel question define subtype(s) family should target; mediate beneficial drug, could be responsible unwanted side effects? One tools, has been developed over last decade elucidate function given gene, generation analysis gene-targeted mice. review summarize progress on identifying individual potential by using genetically modified