Sun Lab

Publications

Lin, Y; Liou, B; Fannin, V; Adler, S; Mayhew, CN; Hammonds, JE; Hu, Y-C; Tchieu, J; Zhang, W; Zhao, X; Kaynak, A; Qi, X; Feldman, RA; Sun, Y. Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies. eLife. 2026; 15.

Zhao, X; Lin, Y; Sun, Y; Liu, C. 029 LINKING PGRN DEFICIENCY AND GBA MUTATION TO LYSOSOMAL DYSFUNCTION IN CHONDROCYTE METABOLISM AND OSTEOARTHRITIS. Osteoarthritis and Cartilage. 2026; 34:s49-s51.

Zhao, X; Fu, W; Lin, Y; Liou, B; Fannin, V; Grabowski, G; Sun, Y; Liu, C. A progranulin derivative blocks the C5a/C5aR1 signaling and mitigates pathology in Gaucher disease. Molecular Genetics and Metabolism. 2026; 147(2):109683.

Sun, Y; Liou, B; Lin, Y; Fannin, V; Kaynak, A; Qi, X. Brain delivery of long-acting enzymes via SapC-DOPS nanocarrier for neuronopathic Gaucher disease. Molecular Genetics and Metabolism. 2026; 147(2):109639.

Comper, F; Miranda, CJ; Liou, B; Dodev, T; Jeyakumar, JM; Canavese, M; Cocita, C; Khoshrou, K; Tiscornia, G; Chisari, E; Dane, AP; Sheridan, R; Nathwani, AC; Corbau, R. FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1. Molecular Therapy. 2025; 33(8):3789-3807.

Lin, Y; Zhao, X; Liou, B; Fannin, V; Zhang, W; Setchell, KDR; Wang, X; Pan, D; Grabowski, GA; Liu, C-J; Sun, Y. Intrinsic link between PGRN and Gba1 D409V mutation dosage in potentiating Gaucher disease. Human Molecular Genetics. 2024; 33(20):1771-1788.

Peng, Y; Liou, B; Lin, Y; Mayhew, CN; Fleming, SM; Sun, Y. iPSC-derived neural precursor cells engineering GBA1 recovers acid β-glucosidase deficiency and diminishes α-synuclein and neuropathology. Molecular Therapy: Methods & Clinical Development. 2023; 29:185-201.

Lin, Y; Zhao, X; Liou, B; Fannin, V; Zhang, W; Liu, Y; Wang, X; Pan, D; Grabowski, G; Liu, C; Sun, Y. Earlier-onset, more severe neurodegeneration in PGRN KO mice with a decreased dose of D409V Gba1. Molecular Genetics and Metabolism. 2023; 138(2):107207.

Zhao, X; Lin, Y; Liou, B; Zhang, W; Liu, C; Sun, Y. A brain penetrant progranulin-derived biologic protects against neuronopathic Gaucher disease. Molecular Genetics and Metabolism. 2023; 138(2):107367.

Zhao, X; Lin, Y; Liou, B; Fu, W; Jian, J; Fannie, V; Zhang, W; Setchell, KDR; Grabowski, GA; Sun, Y; Liu, C-J. PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects, GBA1 mutation-associated pathologies and diseases. Proceedings of the National Academy of Sciences of the United States of America. 2023; 120(1):e2210442120.

Shibuya, Y; Kumar, KK; Mader, MM-D; Yoo, Y; Ayala, LA; Zhou, M; Mohr, MA; Neumayer, G; Kumar, I; Yamamoto, R; Heppner, FL; Wyss-Coray, T; Südhof, TC; Wernig, M. Treatment of a genetic brain disease by CNS-wide microglia replacement. Science Translational Medicine. 2022; 14(636):eabl9945.

Zhao, X; Hasan, S; Liou, B; Lin, Y; Sun, Y; Liu, C. Analysis of the Biomarkers for Neurodegenerative Diseases in Aged Progranulin Deficient Mice. International Journal of Molecular Sciences. 2022; 23(2).

Flunkert, S; Hutter-Paier, B; Sun, Y; Kehr, J. Editorial: Neuronopathic lysosomal storage diseases - specific neuronal characteristics and therapeutic approaches. Frontiers in Molecular Neuroscience. 2022; 15:1078804.