UCLA

Neurodegenerative diseases such as Parkinson’s and Alzheimer’s affect millions of people and are uniformly untreatable. A significant unmet need exists for faster and higher throughput approaches to modeling neurodegenerative diseases and identifying candidate small molecule therapies. Herein, I present a new chemical biology approach to identifying small molecules candidates for modeling and treating neurodegenerative diseases, using pontocerebellar hypoplasia type 1B and amyotrophic lateral sclerosis as test cases. The approach combines in silico rational design, in vitro biophysical testing, and in vivo assays in zebrafish to yield a powerful discovery platform for small molecule models and therapies for neurodegenerative disease.