Mervyn J. Monteiro, PhD
Generation and characterization of ubiquilin-2 transgenic mice models of ALS
Ubiquilin-2 mutations have been shown to cause ALS. To understand the molecular mechanisms by which the mutations cause disease we propose to generate and characterize transgenic mice carrying select human ubiquilin-2 mutations that we have found affect the synthesis, folding and degradation of proteins. The mice will be characterized using a battery of biochemical, pathological and behavioral examinations. Once developed these mice will provide useful models to test and validate candidate approaches to slow and/or halt ALS.