講演情報
[NFS-02-2]Silencing FUS and Other Rare Genetic Forms of ALS
Neil A. Shneider (Columbia University)
Dr. Shneider is the Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University. He is an investigator in the Center for Motor Neuron Biology and Disease where his lab focuses on the study of models and mechanisms of ALS and the development of novel therapeutics for ALS and related disorders. Dr. Shneider worked with Ionis Pharmaceuticals to develop ION363 (jacifusen), an antisense oligonucleotide (ASO) for ALS patients with rare mutations in the FUsed in Sarcoma (FUS) gene. Dr. Shneider is a graduate of Harvard College and earned his M.D. and Ph.D. degrees at the Columbia University College of Physicians and Surgeons. In partnership with n-Lorem and Columbia University, Dr. Shneider founded Silence ALS, an initiative to develop ASOs for ALS patients with ultra-rare, pathogenic mutations in ALS genes. Dr. Shneider was co-chair of the Translating Fundamental Research into Potential ALS Therapies Working Group for the NIH ALS Strategic Planning Workshop, and is a multi-PI for the Access for ALL in ALS Consortium (ALL ALS), a community of 35 research sites across the United States conducting a combined longitudinal natural history study and biomarker collection study for ALS.