Search Results - "Tiger, Pascale M. N."

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

“…Significance Mucopolysaccharidosis type IIIB (MPS IIIB) is a devastating and currently untreatable disease affecting mainly the brain. The cause is lack of the…”
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Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B by Grover, Anita, Crippen-Harmon, Danielle, Nave, Lacey, Vincelette, Jon, Wait, Jill C. M., Melton, Andrew C., Lawrence, Roger, Brown, Jillian R., Webster, Katherine A., Yip, Bryan K., Baridon, Brian, Vitelli, Catherine, Rigney, Sara, Christianson, Terri M., Tiger, Pascale M. N., Lo, Melanie J., Holtzinger, John, Shaywitz, Adam J., Crawford, Brett E., Fitzpatrick, Paul A., LeBowitz, Jonathan H., Bullens, Sherry, Aoyagi-Scharber, Mika, Bunting, Stuart, O’Neill, Charles A., Pinkstaff, Jason, Bagri, Anil

“…BMN 250 is being developed as enzyme replacement therapy for Sanfilippo type B, a primarily neurological rare disease, in which patients have deficient…”
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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice by Aoyagi-Scharber, Mika, Crippen-Harmon, Danielle, Lawrence, Roger, Vincelette, Jon, Yogalingam, Gouri, Prill, Heather, Yip, Bryan K, Baridon, Brian, Vitelli, Catherine, Lee, Amanda, Gorostiza, Olivia, Adintori, Evan G, Minto, Wesley C, Van Vleet, Jeremy L, Yates, Bridget, Rigney, Sara, Christianson, Terri M, Tiger, Pascale M N, Lo, Melanie J, Holtzinger, John, Fitzpatrick, Paul A, LeBowitz, Jonathan H, Bullens, Sherry, Crawford, Brett E, Bunting, Stuart

“…Sanfilippo syndrome type B (mucopolysaccharidosis IIIB), caused by inherited deficiency of α- -acetylglucosaminidase (NAGLU), required for lysosomal…”
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Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction by Magat, Jenna, Jones, Samantha, Baridon, Brian, Agrawal, Vishal, Wong, Hio, Giaramita, Alexander, Mangini, Linley, Handyside, Britta, Vitelli, Catherine, Parker, Monica, Yeung, Natasha, Zhou, Yu, Pungor, Erno, Slabodkin, Ilya, Gorostiza, Olivia, Aguilera, Allora, Lo, Melanie J., Alcozie, Saida, Christianson, Terri M., Tiger, Pascale M.N., Vincelette, Jon, Fong, Sylvia, Gil, Geuncheol, Hague, Chuck, Lawrence, Roger, Wendt, Daniel J., Lebowitz, Jonathan H., Bunting, Stuart, Bullens, Sherry, Crawford, Brett E., Roy, Sushmita M., Woloszynek, Josh C.

“…Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disorder caused by N-sulfoglucosamine sulfohydrolase (SGSH) deficiency. SGSH removes the…”
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New building blocks for the assembly of sequence selective molecular zippers by Hunter, Christopher A, Jones, Philip S, Tiger, Pascale M N, Tomas, Salvador

“…Synthetic H-bonded molecular zippers contain no sequence information that can be used to engineer the selective binding interactions characteristic of…”
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