Structural Biology and Biochemistry Research Theme Seminar

A Possible Strategy for the Treatment of Batten’s Disease: Recovery of Lysosome Function 

Abstract

Batten’s Disease is a rare hereditary neurodegenerative in children. Because of its rarity, pharmaceutical companies are not particularly interested in finding a cure. Any cure would not be profitable. Such diseases are generally referred to as “orphan diseases”. However, a diagnosis of Batten’s disease is devastating for the family of any child afflicted. There are many forms of the disease, but all are caused by lysosomal dysfunction, which results in the steady build up of waste material within cells. Because they’re not regenerated during a person’s lifespan, neurons are particularly in danger. The disease progression is characterised by the development of blindness, followed later by the onset of seizures and an early death, typically in the 20’s. The development of an understanding of the disease would not only help its sufferers, but provide valuable information on a crucial cell function, i.e., how do cells solve the problem of sustainability and recycle their own waste? In this seminar the speaker won’t provide the answer to this question, but he will describe how biophysical measurements are helping to provide some insights into the molecular physiology of lysosome function and the pathology of Batten’s disease. 

Biography

Associate Professor Ronald Clarke graduated from the University of Adelaide in Physical Chemistry in 1986. Because he is a Germanophile and was lucky enough to obtain a scholarship from the Alexander von Humboldt Foundation, in 1987 he moved from Adelaide to Constance, on Lake Constance on the border to Switzerland, to work in the Biophysics Department of the University of Constance. This where he started working on membrane proteins, an example of which is the CLN3 protein, Battenin. Subsequently, he worked at the University of East Anglia, Norwich, in the UK, and then for almost 10 years within the Max-Planck-Society, first in Berlin at the Fritz-Haber-Institute and then in Frankfurt am Main at the Max-Planck-Institute of Biophysics. In 1999 he returned to Australia and took up his current position as a member of academic staff in the School of Chemistry at the University of Sydney. He is now an associate professor within the School. At the same time, he is also the University’s DAAD Ambassador and earlier this year he was appointed as Secretary-General of the International Union of Pure and Applied Biophysics.