Philip C. Wong
Professor - Pathology Johns Hopkins University
Dr. Philip Wong is a professor of pathology and neuroscience at the Johns Hopkins University School of Medicine. His research focuses on understanding the molecular mechanisms of neurodegenerative disorders, such as Alzheimer’s disease (AD) and amyotrophic lateral sclerosis (ALS) – frontotemporal dementia (FTD).
Dr. Wong’s team takes a molecular/cellular approach, including transgenic, gene targeting, and RNAi strategies in mice, to develop models that facilitate their understanding of pathogenesis of AD and ALS-FTD as well as the identification and validation of novel targets for mechanism-based therapeutics.
He received his undergraduate degree in biochemistry and his Ph.D. in biochemistry and molecular biology from the University of Western Ontario in Canada. He completed a postdoctoral fellowship in cellular and molecular biology at the Johns Hopkins University School of Medicine in the Department of Biological Chemistry. Dr. Wong joined the Johns Hopkins faculty in 1994.
Dr. Wong is a member of the Society of Neuroscience and a member of the editorial board for Molecular Neurodegeneration. Dr. Wong’s work has been recognized with the 2004 Zenith Fellow’s Award from the Alzheimer’s Association and the 2007 MetLife Foundation Award for Medical Research in AD.
Seminars
- First in vivo evidence that ASO-mediated restoration of UNC13A cryptic exon function can modify disease-relevant biology, validating UNC13A as a therapeutically actionable target in ALS
- Why partial restoration of UNC13A may be sufficient to move the needle clinically, and how to think about biological sufficiency versus complete target correction
- The critical importance of early intervention, with data supporting delivery before irreversible neuronal loss to maximise therapeutic benefit
- The need for a companion biomarker strategy to guide patient selection and timing in clinical trials, ensuring the right patients receive treatment at the right stage
- What are the earliest cellular events that trigger TDP-43 mislocalization and aggregation, and how do these upstream mechanisms vary across ALS subtypes?
- How do general protein homeostasis pathways, including nuclear import/export and stress response mechanisms, influence TDP-43 pathology?
- Which cryptic exons, epigenetic changes, or C9orf72-related mechanisms are likely contributors to disease initiation, and how should these inform target prioritization?
- How can we improve ALS model systems to better reflect the physiological context of TDP-43 misfolding and aggregate formation?
- Moving beyond restoring TDP-43 function, what strategies can drug developers adopt to intervene at the root cause of aggregate pathology?
