Prof. Aaron Gitler, Ph.D., is the Stanford Medicine Basic Science Professor in the Department of Genetics at Stanford University. He received his B.S. degree from Penn State University and did his PhD studies on cardiovascular development in the laboratory of Dr. Jonathan Epstein at the University of Pennsylvania. Then he performed his postdoctoral training with Dr. Susan Lindquist at the Whitehead Institute for Biomedical Research and MIT. In 2007, he established his laboratory at the University of Pennsylvania and moved to Stanford in 2012. His laboratory has been using a combination of yeast and human genetics approaches to investigate pathogenic mechanisms of ALS. His laboratory has made several fundamental discoveries into neurodegenerative disease mechanisms. These discoveries include discovery of modifiers of aggregation and cytotoxicity of the FTD/ALS disease protein TDP-43, a mechanism to explain how FTD/ALS-linked TDP-43 mutations affect the protein and contribute to disease, and the discovery of novel genetic contributors to human FTD and ALS, including mutations in the ataxin-2 gene as one of the most common genetic risk factors for ALS and a role of cryptic splicing of UNC13A and other synaptic protein encoding genes as a mechanism in ALS and FTD. Gitler’s work has helped to uncover unexpected and novel therapeutic targets for ALS, including preclinical studies of ataxin-2 as a therapeutic target for sporadic ALS, demonstrating that reduction of ataxin-2 levels markedly extends lifespan in TDP-43 transgenic mice. These efforts have provided the foundation for an ongoing clinical trial in human ALS patients to test ataxin-2 targeting antisense oligonucleotides as a therapy.