| dc.description.abstract | Technology is continually improved to overcome particular obstacles and then
often proves useful in an array of applications. Biological understanding of both human
physiology and the disease of interest allows for new approaches to be developed. In the
case of Mycobacterium tuberculosis (Mtb), the pathogen has become extremely drug
resistant, requiring new therapies, combination therapies, that are targeted at newly
discovered vulnerabilities. Cancer, a leading cause of death, requires sophisticated
therapies that are based on a better understanding of the biology behind such a diverse
disease. Cancers that are inherently resistant to standard therapies, and those that result in
relapse, require new treatment options to provide patients with a better chance of survival
and improved quality of life. Other hereditary diseases, such as Menkes disease, are rare
but result in an extremely poor quality of life as well as early death and have no effective
therapy.
Genetic and physiological characterization, as well as subsequent target protein
examination when applicable, provide insight into possible approaches for effective
therapy. Advancements in treating other diseases, or failures in the case of Menkes, often
provide useful information applicable to other diseases as well. As new reporter systems
are developed for characterization or disease therapy, they also may provide the
information necessary to overcome hurdles experienced when working with other, even
completely different diseases.
Any particular assay or therapy will have limitations in throughput and information
gained. As assays get more complex, the throughput decreases proportionally. In this text
are three examples employing a base understanding of disease vulnerabilities, along with
modern investigative tools to develop an efficacy assay that will provide evidence that a
particular therapy warrants further development or that future research should focus on
alternative therapies. This work focuses on developing and employing assays that
maximize throughput to minimize the chance that limited resources will prevent an
effective therapy from progressing into clinical trials. | |
