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Always kind, respectful, and approachable.
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Ying Sun, PhD, serves as Professor-Affiliate in the Department of Pediatrics within the University of Cincinnati College of Medicine and as a faculty member in the Division of Human Genetics at Cincinnati Children's Hospital Medical Center. She obtained her PhD in Pharmacology from the University of Cincinnati in 1992, followed by a postdoctoral fellowship in Human Genetics at Cincinnati Children's Hospital Medical Center, completed in 1996. Her academic career has been dedicated to advancing the understanding of lysosomal storage diseases, particularly focusing on their molecular pathogenesis and therapeutic interventions. Sun's research encompasses glycosphingolipid metabolism in neurodegenerative contexts, elucidating connections between Gaucher disease and Parkinson’s disease via lipidomics and transcriptomics. Her laboratory utilizes brain organoid models, pharmaceutical small molecule therapies, and investigates the therapeutic efficacy of induced pluripotent stem cell-derived neural progenitor cells to enhance lysosomal acid β-glucosidase activity in Parkinson’s models. Additionally, she explores progranulin's role as a modulator of acid β-glucosidase for potential Gaucher disease treatments. This work is supported by funding from the National Institutes of Health, foundations, and industry-sponsored programs.
Sun has made significant contributions through numerous peer-reviewed publications in prestigious journals. Notable works include 'FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1' (Molecular Therapy, 2025), 'Intrinsic link between PGRN and Gba1 D409V mutation dosage in potentiating Gaucher disease' (Human Molecular Genetics, 2024), 'iPSC-derived neural precursor cells engineering GBA1 recovers acid β-glucosidase deficiency and diminishes α-synuclein and neuropathology' (Molecular Therapy: Methods & Clinical Development, 2023), 'PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects, GBA1 mutation-associated pathologies and diseases' (Proceedings of the National Academy of Sciences, 2023), 'Earlier-onset, more severe neurodegeneration in PGRN KO mice with a decreased dose of D409V Gba1' (Molecular Genetics and Metabolism, 2023), and 'A brain penetrant progranulin-derived biologic protects against neuronopathic Gaucher disease' (Molecular Genetics and Metabolism, 2023). She has also contributed editorially, co-authoring 'Editorial: Neuronopathic lysosomal storage diseases - specific neuronal characteristics and therapeutic approaches' (Frontiers in Molecular Neuroscience, 2022). These efforts underscore her impact on developing translational therapies for neurodegenerative lysosomal storage disorders.
