Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a relentlessly progressive, fatal neurodegenerative disease. Patients with ALS suffer from degeneration of motor neurons in the brain and spinal cord, leading to progressive muscular weakness, culminating in death due to respiratory paralysis, typically 2-5 years after onset. Unfortunately, no effective treatment is currently available.
While most forms of ALS are sporadic and idiopathic (sALS), ~10% of cases are inherited in a Mendelian fashion and are designated familial ALS (fALS). My research interest is to characterize the functions of proteins involved in fALS with the aim of targeting them for drug design. The goal of my research is to study the molecular mechanism of ALS, and to utilize the information for new therapeutics development. To achieve this goal, interdisciplinary approaches involved in several research areas will be utilized, including molecular and cellular biology, biochemistry and structure biology, large scale compound screening, and structure based drug design.
