Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction

Authors: 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.

Source: In Journal of Biological Chemistry December 2022 298(12)

Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice

Authors: Chen, Joseph C., Luu, Amanda R., Wise, Nathan, Angelis, Rolando De, Agrawal, Vishal, Mangini, Linley, Vincelette, Jon, Handyside, Britta, Sterling, Harry, Lo, Melanie J., Wong, Hio, Galicia, Nicole, Pacheco, Glenn, Van Vleet, Jeremy, Giaramita, Alexander, Fong, Sylvia, Roy, Sushmita M., Hague, Chuck, Lawrence, Roger, Bullens, Sherry, Christianson, Terri M., d'Azzo, Alessandra, Crawford, Brett E., Bunting, Stuart, LeBowitz, Jonathan H., Yogalingam, Gouri

Source: In Journal of Biological Chemistry 25 September 2020 295(39):13532-13555

Intermittent enzyme replacement therapy with recombinant human β-galactosidase prevents neuraminidase 1 deficiency

Authors: Luu, Amanda R., Wong, Cara, Agrawal, Vishal, Wise, Nathan, Handyside, Britta, Lo, Melanie J., Pacheco, Glenn, Felix, Jessica B., Giaramita, Alexander, d'Azzo, Alessandra, Vincelette, Jon, Bullens, Sherry, Bunting, Stuart, Christianson, Terri M., Hague, Charles M., LeBowitz, Jonathan H., Yogalingam, Gouri ^*

Source: In Journal of Biological Chemistry 25 September 2020 295(39):13556-13569

Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B

Authors: Prill, Heather, Luu, Amanda, Yip, Bryan, Holtzinger, John, Lo, Melanie J., Christianson, Terri M., Yogalingam, Gouri, Aoyagi-Scharber, Mika, LeBowitz, Jonathan H., Crawford, Brett E., Lawrence, Roger

Source: In Molecular Therapy - Methods & Clinical Development 13 September 2019 14:56-63

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

Authors: 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

Source: Drug delivery and translational research. 10(2):425-439

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100164283678.0x000001&indx=1&recIds=ETOCvdc_100164283678.0x000001

Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

Authors: 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

Source: In Molecular Therapy - Methods & Clinical Development 15 September 2017 6:43-53

Lipoprotein Receptor Binding, Cellular Uptake, and Lysosomal Delivery of Fusions between the Receptor-associated Protein (RAP) and α-l-Iduronidase or Acid α-Glucosidase

Authors: Prince, William S. ^‡, McCormick, Lynn M. ^§, Wendt, Dan J. ^‡, Fitzpatrick, Paul A. ^‡, Schwartz, Keri L. ^‡, Aguilera, Allora I. ^‡, Koppaka, Vishwanath ^‡, Christianson, Terri M. ^‡, Vellard, Michel C. ^‡, Pavloff, Nadine ^‡, Lemontt, Jeff F. ^‡, Qin, Minmin ^‡, Starr, Chris M. ^‡, Bu, Guojun ^{§, ¶}, Zankel, Todd C. ^{‡, ∥}

Source: In Journal of Biological Chemistry 13 August 2004 279(33):35037-35046

Correction: Intermittent enzyme replacement therapy prevents Neu1 deficiency

Authors: Luu, Amanda R., Wong, Cara, Agrawal, Vishal, Wise, Nathan, Handyside, Britta, Lo, Melanie J., Pacheco, Glenn, Felix, Jessica B., Giaramita, Alexander, d'Azzo, Alessandra, Vincelette, Jon, Bullens, Sherry, Bunting, Stuart, Christianson, Terri M., Hague, Charles M., LeBowitz, Jonathan H., Yogalingam, Gouri

Source: In Journal of Biological Chemistry 13 November 2020 295(46):15794-15794

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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

Overexpression of inactive arylsulphatase mutants and in vitro activation by light-dependent oxidation with vanadate

Authors: CHRISTIANSON, Terri M., STARR, Chris M., ZANKEL, Todd C.

Source: Biochemical Journal; September 2004, Vol. 382 Issue: 2 p581-587, 7p