[NFS-02] Neuroscience Frontier Symposium 02

[NFS-02-1] Designer DNA drug therapy for ALS and other TDP-43 proteinopathies

Don Cleveland (University of California, San Diego)

Don Cleveland is Professor and Chair of Cellular and Molecular Medicine at UC San Diego He has been elected to the U.S. National Academy of Sciences and National Academy of Medicine. He has identified principles of genome instability in cancer, demonstrating that single chromosome missegregation can trigger repeated chromosome shattering (chromothripsis) that initiates and drives genome evolution in cancer. For this work, in 2019 he became the 15th recipient of India’s Genome Valley Excellence Award. In neurosciences, he purified and characterized the first microtubule associated protein - tau - which misassembles in neurons in Alzheimer’s disease and chronic brain injury. He uncovered mechanisms underlying the major genetic forms of Amyotrophic Lateral Sclerosis (ALS) and developed “designer DNA drugs” for silencing disease-causing genes responsible for the major diseases of the nervous system, with clinical trials now ongoing in ALS, Parkinson’s, and Alzheimer’s diseases. For his efforts in neurosciences, he received the 2018 Breakthrough Prize in Life Sciences, the 2019 Sean M. Healey International Prize for Innovation in ALS, the 2022 Lalji & Family ALS Endowed Award for Innovative Healing, the 2022 E.B. Wilson Medal, and the 2023 Rainwater Prize.