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

Authors: 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.

Source: In Molecular Therapy - Methods & Clinical Development 16 March 2018 8:42-51

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

Authors: 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.

Source: In Experimental Neurology November 2015 273:1-10

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

Authors: Kan, Shih-hsin, Aoyagi-Scharber, Mika, Le, Steven Q., Vincelette, Jon, Ohmi, Kazuhiro, Bullens, Sherry, Wendt, Daniel J., Christiansen, Terri M., Tiger, Pascale M. N., Brown, Julian 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.

Source: Proceedings of the National Academy of Sciences of the United States of America, 2014 Oct . 111(41), 14870-14875.

Access URL: https://www.jstor.org/stable/43190169

Characterization on the cellular distribution of enzyme replacement therapy (ERT) under the influence of humoral immune response in MPS I mouse model

Authors: Sanghez, Valentina, Kan, Shih-hsin, Le, Steven Q, Clarke, Don, Vondrak, Kristen N, Vera, Moin, Iacovino, Michelina, Sands, Mark, Dickson, Patti

Source: Molecular genetics and metabolism. 120(1/2):S118-S118

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100052510546.0x000001&indx=1&recIds=ETOCvdc_100052510546.0x000001

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

Authors: Shih-hsin Kana, Aoyagi-Scharber, Mika, Lea, Steven Q., Jon Vincelette, 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., Zhi Chen, Hague, Chuck M., Woloszynek, Josh C., Cheung, Diana S.

Source: Proceedings of the National Academy of Sciences of the United States of America; 10/14/2014, Vol. 111 Issue 41, p14870-14875, 6p

Subject Terms: SOMATOMEDIN A, INSULIN-like growth factor-binding proteins, CARRIER proteins, MUCOPOLYSACCHARIDOSIS treatment, CARBOHYDRATE metabolism disorders