Methods and compositions for development of drug screening procedures and diagnostic tools

Abstract

This invention defines novel research methodology for use in (a) monitoring the ongoing status of the physiological expression of Charcot-Marie-Tooth disease and (b) screening candidate therapeutic drug agents for possible effectiveness. The presence of this neurodegenerative disease can be characterized in part by the expression in cultured fibroblasts obtained from the patient of one or more proteins which are not the product of a defective disease-inducing gene, but which are stress proteins, one or more other proteins modified by conditions of oxidative stress or other disease-related proteins. This candidate drug screening technology offers advantages in terms of (a) providing new research opportunities, (b) cost effectiveness, (c) ability to be used readily on a large scale, (d) ability to generate meaningful data in a comparatively short period of time, and (e) providing an opportunity to obtain information based on direct interaction of a candidate drug and a living tissue disease model.