演者

Boxun Lu, PhD
Professor, School of Life Sciences
Fudan University, Shanghai, China

演題

Drugging the Undruggable: Application of HTRF in Targeted Drug Discovery for Neurodegenerative Disorders

要旨

Classical targeted drug discovery is based on targeting druggable targets, typically kinases and receptors of which the function can be agonized or antagonized. This strategy meets difficulties in neurodegenerative disorders, where the pathological function of the protein causing the disease is unclear, preventing efforts to screen for ‘inhibitors’ of the disease proteins. Although these “neurodegeneration-causing” proteins are not conventional ‘druggable’ targets, we may target them by screening for compound and genetic modifiers of their levels, degradation or conformation to treat the disease. Such screening studies could be achieved by HTRF technology, which provides unique advantages for protein assays. We have successfully used this technology in our targeted drug discovery studies for neurodegenerative disorders. In this talk, I will present some of our recently assay development and screening efforts using the HTRF technology, with a focus on drug discovery for Huntington’s disease (HD), an important monogenetic neurodegenerative disorder.