Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro

بواسطة Tippin, Brigette L., Troitskaya, Larisa, Kan, Shih-hsin, Todd, Amanda K., Le, Steven Q., Dickson, Patricia I.
منشور في 2011

Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice

بواسطة Kan, Shih-hsin, Le, Steven Q., Bui, Quang D., Benedict, Braeden, Cushman, Jesse, Sands, Mark S., Dickson, Patricia I.
الحاوية / القاعدة Behav Brain Res (2016)

Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice

بواسطة Clarke, Don, Pearse, Yewande, Kan, Shih-hsin, Le, Steven Q., Sanghez, Valentina, Cooper, Jonathan D., Dickson, Patricia I., Iacovino, Michelina
الحاوية / القاعدة Mol Ther Methods Clin Dev (2018)

Missense Mutations in the Homeodomain of HOXD13 Are Associated with Brachydactyly Types D and E

بواسطة Johnson, David, Kan, Shih-hsin, Oldridge, Michael, Trembath, Richard C., Roche, Philippe, Esnouf, Robert M., Giele, Henk, Wilkie, Andrew O. M.
منشور في 2003

The Immune Response to Intrathecal Enzyme Replacement Therapy in Mucopolysaccharidosis I Patients

بواسطة Vera, Moin, Le, Steven, Kan, Shih-hsin, Garban, Hermes, Naylor, David, Mlikotic, Anton, Kaitila, Ilkka, Harmatz, Paul, Chen, Agnes, Dickson, Patricia
منشور في 2013

Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts

بواسطة 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.
منشور في 2014

CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness

بواسطة Huang, Jeffrey Y., Kan, Shih-Hsin, Sandfeld, Emilie K., Dalton, Nancy D., Rangel, Anthony D., Chan, Yunghang, Davis-Turak, Jeremy, Neumann, Jon, Wang, Raymond Y.
الحاوية / القاعدة Sci Rep (2020)

Myelin and lipid composition of the corpus callosum in mucopolysaccharidosis type I mice

بواسطة 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.
الحاوية / القاعدة Lipids (2020)

Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome

بواسطة 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.
الحاوية / القاعدة Mol Genet Metab (2021)

A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I

بواسطة 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.
الحاوية / القاعدة Mol Ther Methods Clin Dev (2017)

Intra-articular Enzyme Replacement Therapy with rhIDUA is Safe, Well-Tolerated, and Reduces Articular GAG Storage in the Canine Model of Mucopolysaccharidosis Type I

بواسطة Wang, Raymond Y, Aminian, Afshin, McEntee, Michael F, Kan, Shih-Hsin, Simonaro, Calogera M, Lamanna, William, Lawrence, Roger, Ellinwood, N. Matthew, Guerra, Catalina, Le, Steven Q, Dickson, Patricia I, Esko, Jeffrey D
منشور في 2014

Genomic Screening of Fibroblast Growth-Factor Receptor 2 Reveals a Wide Spectrum of Mutations in Patients with Syndromic Craniosynostosis

بواسطة Kan, Shih-hsin, Elanko, Navaratnam, Johnson, David, Cornejo-Roldan, Laura, Cook, Jackie, Reich, Elsa W., Tomkins, Susan, Verloes, Alain, Twigg, Stephen R. F., Rannan-Eliya, Sahan, McDonald-McGinn, Donna M., Zackai, Elaine H., Wall, Steven A., Muenke, Maximilian, Wilkie, Andrew O. M.
منشور في 2002

Features of Brain MRI in Dogs with Treated and Untreated Mucopolysaccharidosis Type I

بواسطة Vite, Charles H, Nestrasil, Igor, Mlikotic, Anton, Jens, Jackie K, Snella, Elizabeth M, Gross, William, Shapiro, Elsa G, Kovac, Victor, Provenzale, James M, Chen, Steven, Le, Steven Q, Kan, Shih-hsin, Banakar, Shida, Wang, Raymond Y, Haskins, Mark E, Ellinwood, N Matthew, Dickson, Patricia I
منشور في 2013

A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I

بواسطة 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
الحاوية / القاعدة Mol Ther Methods Clin Dev (2015)

Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I

بواسطة 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.
الحاوية / القاعدة Exp Neurol (2015)

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I

بواسطة 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
منشور في 2010

Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

بواسطة Kan, Shih-hsin, Aoyagi-Scharber, Mika, Le, Steven Q., Vincelette, Jon, Ohmi, Kazuhiro, Bullens, Sherry, Wendt, Daniel J., Christianson, Terri M., Tiger, Pascale M. N., Brown, Jillian R., Lawrence, Roger, Yip, Bryan K., Holtzinger, John, Bagri, Anil, Crippen-Harmon, Danielle, Vondrak, Kristen N., Chen, Zhi, Hague, Chuck M., Woloszynek, Josh C., Cheung, Diana S., Webster, Katherine A., Adintori, Evan G., Lo, Melanie J., Wong, Wesley, Fitzpatrick, Paul A., LeBowitz, Jonathan H., Crawford, Brett E., Bunting, Stuart, Dickson, Patricia I., Neufeld, Elizabeth F.
منشور في 2014