Maurice Wilkins Centre Research Seminar
Associate Professor David Sleat
Lysosomal Proteins, Proteomics and Disease
To gain insights into the normal biology of the lysosome and to understand the mo-lecular bases for disorders rooted in disrupted lysosomal function, our laboratory has made extensive use of proteomic methods to characterize the components of this organelle. Proteomic approaches have allowed us to identify genetic bases for two human lysosomal storage diseases, late-infantile neuronal ceroid lipofuscinosis (LINCL) and Niemann-Pick C type 2, and allowed us to identify defects in a number of cases of lysosomal disease of unknown etiology. More recently, we have applied proteomic methods to address a pressing need for useful biomarkers to measure disease progression and response to therapy in LINCL and related diseases. I will provide an update on our progress in this area and also describe our recent efforts to extend the proteomic characterization of the lysosome to create a comprehen-sive compartmental map of the mammalian cell.