Publications

Dai, M; Lin, Y; El-Amouri, SS; Kohls, M; Pan, D. Comprehensive evaluation of blood-brain barrier-forming micro-vasculatures: Reference and marker genes with cellular composition. PloS one. 2018; 13:e0197379.

El-Amouri, SS; Dai, M; Han, J; Brady, RO; Pan, D. Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase. Molecular Therapy. 2014; 22:2028-2037.

Dai, M; Han, J; El-Amouri, SS; Brady, RO; Pan, D. Platelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler syndrome. Proceedings of the National Academy of Sciences of USA. 2014; 111:2680-2685.

Wang, D; El-Amouri, SS; Dai, M; Kuan, C; Hui, DY; Brady, RO; Pan, D. Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier. Proceedings of the National Academy of Sciences of USA. 2013; 110:2999-3004.

Pan, D; Kalfa, TA; Wang, D; Risinger, M; Crable, S; Ottlinger, A; Chandra, S; Mount, DB; Huebner, CA; Franco, RS; Joiner, CH. K-Cl cotransporter gene expression during human and murine erythroid differentiation. The Journal of biological chemistry. 2011; 286:30492-30503.

Wang, D; Zhang, W; Kalfa, TA; Grabowski, G; Davies, S; Malik, P; Pan, D. Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome. Proceedings of the National Academy of Sciences of USA. 2009; 106:19958-19963.

Worsham, DN; Schuesler, T; von Kalle, C; Pan, D. In vivo gene transfer into adult stem cells in unconditioned mice by in situ delivery of a lentiviral vector. Molecular Therapy. 2006; 14:514-524.

Pan, D; Gunther, R; Duan, WM; Wendell, S; Kaemmerer, W; Kafri, T; Verma, IM; Whitley, CB. Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow. Molecular Therapy. 2002; 6:19-29.

Pan, D; Shankar, R; Stroncek, DF; Whitley, CB. Combined ultrafiltration-transduction in a hollow-fiber bioreactor facilitates retrovirus-mediated gene transfer into peripheral blood lymphocytes from patients with mucopolysaccharidosis type II. Human Gene Therapy. 1999; 10:2799-2810.

Zhang, Z; Wang, X; Lin, Y; Pan, D. A multifaceted evaluation of microgliosis and differential cellular dysregulations of mTOR signaling with fluctuating lysosome function in neuronopathic Gaucher disease. Molecular Genetics and Metabolism. 2023; 138:107366.

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; 138:107207.

Boateng-Antwi, MK A; Lin, Y; Ren, S; Wang, X; Pan, D. New function of a well-known promoter: Enhancer activity of minimal CMV promoter enables efficient dual-cassette transgene expression. The Journal of Gene Medicine. 2021; 23:e3380.

Lin, Y; Wang, X; Rose, KP; Dai, M; Han, J; Xin, M; Pan, D. miR-143 Regulates Lysosomal Enzyme Transport across the Blood-Brain Barrier and Transforms CNS Treatment for Mucopolysaccharidosis Type I. Molecular Therapy. 2020; 28:2161-2176.