Decoding Neurodegeneration Using iPSCs, CRISPR, and ‘omics

Michael Ward, MD, PhD
Senior Investigator, NINDS, National Institutes of Health
Co-Director, iPSC Neurodegenerative Disease Initiative

Dr. Ward is a Neurologist and Senior Investigator at the National Institutes of Health (NIH). His research focuses on understanding basic mechanisms of age-related neurodegenerative disorders to enable development of next-generation precision therapeutics. Over the past decade, his lab has contributed substantially to our knowledge about underlying causes of neurodegeneration. These include 1) co-discovery of UNC13A mis-splicing in amyotrophic lateral sclerosis (ALS), which sparked development of new ASO-based therapies for this disorder, 2) protein-based biomarkers of TDP-43 dysfunction, a critical unmet need in the neurodegeneration field, and 3) basic mechanisms by which RNAs traffic in neurons via lysosomal hitchhiking. He also co-directs the iPSC Neurodegenerative Disease Initiative (iDNI), a public-private partnership that has grown to become the largest genetically engineered iPSC repository to date, and has distributed cell lines to nearly 1000 labs worldwide since its launch in 2022.