Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts
Kan, Shih-hsin, Troitskaya, Larisa A, Sinow, Carolyn S, Haitz, Karyn, Todd, Amanda K, Di Stefano, Ariana, Le, Steven Q, Dickson, Patricia I, Tippin, Brigette L
Published in Biochemical journal (01.03.2014)
Published in Biochemical journal (01.03.2014)
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Journal Article
Combining angiotensin receptor blockade and enzyme replacement therapy for vascular disease in mucopolysaccharidosis type I
Hurt, Sarah C., Vera, Moin U., Le, Steven Q., Kan, Shih-hsin, Bui, Quang, Dickson, Patricia I.
Published in Molecular genetics and metabolism reports (01.03.2024)
Published in Molecular genetics and metabolism reports (01.03.2024)
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Journal Article
Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease
Pearse, Yewande, Clarke, Don, Kan, Shih-hsin, Le, Steven Q., Sanghez, Valentina, Luzzi, Anna, Pham, Ivy, Nih, Lina R., Cooper, Jonathan D., Dickson, Patricia I., Iacovino, Michelina
Published in Molecular therapy. Methods & clinical development (08.12.2022)
Published in Molecular therapy. Methods & clinical development (08.12.2022)
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Journal Article
Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I
Dickson, Patricia I., Ellinwood, N. Matthew, Brown, Jillian R., Witt, Robert G., Le, Steven Q., Passage, Merry B., Vera, Moin U., Crawford, Brett E.
Published in Molecular genetics and metabolism (01.05.2012)
Published in Molecular genetics and metabolism (01.05.2012)
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Journal Article
A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I
Le, Steven Q, Kan, Shih-Hsin, Clarke, Don, Sanghez, Valentina, Egeland, Martin, Vondrak, Kristen N, Doherty, Terence M, Vera, Moin U, Iacovino, Michelina, Cooper, Jonathan D, Sands, Mark S, Dickson, Patricia I
Published in Molecular therapy. Methods & clinical development (16.03.2018)
Published in Molecular therapy. Methods & clinical development (16.03.2018)
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Journal Article
A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I
Mendez, Daniel C, Stover, Alexander E, Rangel, Anthony D, Brick, David J, Nethercott, Hubert E, Torres, Marissa A, Khalid, Omar, Wong, Andrew Ms, Cooper, Jonathan D, Jester, James V, Monuki, Edwin S, McGuire, Cian, Le, Steven Q, Kan, Shih-Hsin, Dickson, Patricia I, Schwartz, Philip H
Published in Molecular therapy. Methods & clinical development (01.01.2015)
Published in Molecular therapy. Methods & clinical development (01.01.2015)
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Journal Article
Neuropathology of murine Sanfilippo D syndrome
Takahashi, Keigo, Le, Steven Q., Kan, Shih-hsin, Jansen, Matthew J., Dickson, Patricia I., Cooper, Jonathan D.
Published in Molecular genetics and metabolism (01.12.2021)
Published in Molecular genetics and metabolism (01.12.2021)
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Journal Article
Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes
Eisengart, Julie B, Pierpont, Elizabeth I, Kaizer, Alexander M, Rudser, Kyle D, King, Kelly E, Pasquali, Marzia, Polgreen, Lynda E, Dickson, Patricia I, Le, Steven Q, Miller, Weston P, Tolar, Jakub, Orchard, Paul J, Lund, Troy C
Published in Genetics in medicine (01.11.2019)
Published in Genetics in medicine (01.11.2019)
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Journal Article
Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α- N -Acetylglucosamine-6-Sulfatase in Neonatal Mice
Wang, Feng, Moen, Derek R, Sauni, Chelsee, Kan, Shih-Hsin, Li, Shan, Le, Steven Q, Lomenick, Brett, Zhang, Xiaoyi, Ekins, Sean, Singamsetty, Srikanth, Wood, Jill, Dickson, Patricia I, Chou, Tsui-Fen
Published in Molecular pharmaceutics (04.01.2021)
Published in Molecular pharmaceutics (04.01.2021)
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Journal Article
Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study
Chen, Agnes H., Harmatz, Paul, Nestrasil, Igor, Eisengart, Julie B., King, Kelly E., Rudser, Kyle, Kaizer, Alexander M., Svatkova, Alena, Wakumoto, Amy, Le, Steven Q., Madden, Jacqueline, Young, Sarah, Zhang, Haoyue, Polgreen, Lynda E., Dickson, Patricia I.
Published in Molecular genetics and metabolism (01.02.2020)
Published in Molecular genetics and metabolism (01.02.2020)
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Journal Article
Comparison of dermatan sulfate and heparan sulfate concentrations in serum, cerebrospinal fluid and urine in patients with mucopolysaccharidosis type I receiving intravenous and intrathecal enzyme replacement therapy
Zhang, Haoyue, Dickson, Patricia I., Stiles, Ashlee R., Chen, Agnes H., Le, Steven Q., McCaw, Patricia, Beasley, James, Millington, David S., Young, Sarah P.
Published in Clinica chimica acta (01.09.2020)
Published in Clinica chimica acta (01.09.2020)
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Journal Article
Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice
Le, Steven Q., Nestrasil, Igor, Kan, Shih‐hsin, Egeland, Martin, Cooper, Jonathan D., Elashoff, David, Guo, Rong, Tolar, Jakub, Yee, Jennifer K., Dickson, Patricia I.
Published in Lipids (01.11.2020)
Published in Lipids (01.11.2020)
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Journal Article
Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep
Nelvagal, Hemanth R, Eaton, Samantha L, Wang, Sophie H, Eultgen, Elizabeth M, Takahashi, Keigo, Le, Steven Q, Nesbitt, Rachel, Dearborn, Joshua T, Siano, Nicholas, Puhl, Ana C, Dickson, Patricia I, Thompson, Gerard, Murdoch, Fraser, Brennan, Paul M, Gray, Mark, Greenhalgh, Stephen N, Tennant, Peter, Gregson, Rachael, Clutton, Eddie, Nixon, James, Proudfoot, Chris, Guido, Stefano, Lillico, Simon G, Whitelaw, C Bruce A, Lu, Jui-Yun, Hofmann, Sandra L, Ekins, Sean, Sands, Mark S, Wishart, Thomas M, Cooper, Jonathan D
Published in The Journal of clinical investigation (17.10.2022)
Published in The Journal of clinical investigation (17.10.2022)
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Journal Article
Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome
Kan, Shih-hsin, Elsharkawi, Ibrahim, Le, Steven Q., Prill, Heather, Mangini, Linley, Cooper, Jonathan D., Lawrence, Roger, Sands, Mark S., Crawford, Brett E., Dickson, Patricia I.
Published in Molecular genetics and metabolism (01.06.2021)
Published in Molecular genetics and metabolism (01.06.2021)
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Journal Article
Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I
Vera, Moin U., Le, Steven Q., Victoroff, Alla, Passage, Merry B., Brown, Jillian R., Crawford, Brett E., Polgreen, Lynda E., Chen, Agnes H., Dickson, Patricia I.
Published in Molecular genetics and metabolism (01.02.2020)
Published in Molecular genetics and metabolism (01.02.2020)
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Journal Article
Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I
Dickson, Patricia I., Kaitila, Ilkka, Harmatz, Paul, Mlikotic, Anton, Chen, Agnes H., Victoroff, Alla, Passage, Merry B., Madden, Jacqueline, Le, Steven Q., Naylor, David E.
Published in Molecular genetics and metabolism (01.09.2015)
Published in Molecular genetics and metabolism (01.09.2015)
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Journal Article
Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I
Provenzale, James M., Nestrasil, Igor, Chen, Steven, Kan, Shih-hsin, Le, Steven Q., Jens, Jacqueline K., Snella, Elizabeth M., Vondrak, Kristen N., Yee, Jennifer K., Vite, Charles H., Elashoff, David, Duan, Lewei, Wang, Raymond Y., Ellinwood, N. Matthew, Guzman, Miguel A., Shapiro, Elsa G., Dickson, Patricia I.
Published in Experimental neurology (01.11.2015)
Published in Experimental neurology (01.11.2015)
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Journal Article
Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I
Wang, Raymond Y., Aminian, Afshin, McEntee, Michael F., Kan, Shih-Hsin, Simonaro, Calogera M., Lamanna, William C., Lawrence, Roger, Ellinwood, N. Matthew, Guerra, Catalina, Le, Steven Q., Dickson, Patricia I., Esko, Jeffrey D.
Published in Molecular genetics and metabolism (01.08.2014)
Published in Molecular genetics and metabolism (01.08.2014)
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Journal Article
Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I
Dickson, Patricia I., Hanson, Stephen, McEntee, Michael F., Vite, Charles H., Vogler, Carole A., Mlikotic, Anton, Chen, Agnes H., Ponder, Katherine P., Haskins, Mark E., Tippin, Brigette L., Le, Steven Q., Passage, Merry B., Guerra, Catalina, Dierenfeld, Ashley, Jens, Jackie, Snella, Elizabeth, Kan, Shih-hsin, Ellinwood, N. Matthew
Published in Molecular genetics and metabolism (01.10.2010)
Published in Molecular genetics and metabolism (01.10.2010)
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