CNS Tissue-selective Transgenic Animal Mouse Model

Introduction A central nervous system (CNS) specific knockout system has been unavailable thus far due to technical limitations. Recent advances have provided exciting data based on the use a liver-specific promoter to provide deletion of specific genes only in that tissue. This has successfully translated the concept, "tissue-targeted gene function deletion" into practice and could be considered as proof-of-principle data for a tissue-specific knockout system. The next challenge will be developing a CNS tissuespecific knockout system, which will allow evaluation of a large number of CNS drugs and drug candidates that have a low to poor therapeutic index due to very limited tissue availability. Technology description The development of a CNS-specific Cre transgenic model is much needed, specifically ones that target the choroid plexus – the most important brain region for regulating CNS drug disposition. Scientists at the University of Washington have developed methods to identify CNS-specific promoters and use these promoters to provide transgenic models. These models will be useful tools in the evaluation of the role of target proteins in enhancing or limiting the delivery of drug into CNS
(crossing the blood brain barrier) and in mediating both therapeutic and toxicologic responses. Successful development of such a platform will change the paradigms of how we elucidate the functional role of any molecule (receptor, transporter or enzyme) central to CNS delivery and disposition. Business opportunity CNS-specific mouse knockouts will be extremely useful for discovery and evaluation of drug candidates. Stage of development This technology is currently being developed. Intellectual property position The UW is currently reviewing this technology for worldwide patent protection.

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