A breach in the blood-brain barrier

بواسطة LeBowitz, Jonathan H.
منشور في 2005

Utilizing ExAC to assess the hidden contribution of variants of unknown significance to Sanfilippo Type B incidence

بواسطة Clark, Wyatt T., Yu, G. Karen, Aoyagi-Scharber, Mika, LeBowitz, Jonathan H.
الحاوية / القاعدة PLoS One (2018)

A Negative Regulatory Element Controls mRNA Abundance of the Leishmania mexicana Paraflagellar Rod Gene PFR2†

بواسطة Mishra, Krishna K., Holzer, Timothy R., Moore, Landon L., LeBowitz, Jonathan H.
منشور في 2003

Coordinate regulation of a family of promastigote-enriched mRNAs by the 3′UTR PRE element in Leishmania mexicana

بواسطة Holzer, Timothy R., Mishra, Krishna K., LeBowitz, Jonathan H., Forney, James D.
منشور في 2007

Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice

بواسطة LeBowitz, Jonathan H., Grubb, Jeffrey H., Maga, John A., Schmiel, Deborah H., Vogler, Carole, Sly, William S.
منشور في 2004

Cellular Uptake and Delivery of Myeloperoxidase to Lysosomes Promote Lipofuscin Degradation and Lysosomal Stress in Retinal Cells

بواسطة Yogalingam, Gouri, Lee, Amanda R., Mackenzie, Donald S., Maures, Travis J., Rafalko, Agnes, Prill, Heather, Berguig, Geoffrey Y., Hague, Chuck, Christianson, Terri, Bell, Sean M., LeBowitz, Jonathan H.
الحاوية / القاعدة J Biol Chem (2017)

BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pat...

بواسطة Yogalingam, Gouri, Luu, Amanda R., Prill, Heather, Lo, Melanie J., Yip, Bryan, Holtzinger, John, Christianson, Terri, Aoyagi-Scharber, Mika, Lawrence, Roger, Crawford, Brett E., LeBowitz, Jonathan H.
الحاوية / القاعدة PLoS One (2019)

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

بواسطة 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
الحاوية / القاعدة Mol Ther Methods Clin Dev (2019)

Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice

بواسطة Maga, John A., Zhou, Jianghong, Kambampati, Ravi, Peng, Susan, Wang, Xu, Bohnsack, Richard N., Thomm, Angela, Golata, Sarah, Tom, Peggy, Dahms, Nancy M., Byrne, Barry J., LeBowitz, Jonathan H.
منشور في 2013

Characterization of glycan substrates accumulating in GM1 Gangliosidosis

بواسطة Lawrence, Roger, Van Vleet, Jeremy L., Mangini, Linley, Harris, Adam, Martin, Nathan, Clark, Wyatt, Chandriani, Sanjay, LeBowitz, Jonathan H., Giugliani, Roberto, d'Azzo, Alessandra, Yogalingam, Gouri, Crawford, Brett E.
الحاوية / القاعدة Mol Genet Metab Rep (2019)

Identifying therapeutic drug targets using bidirectional effect genes

بواسطة Estrada, Karol, Froelich, Steven, Wuster, Arthur, Bauer, Christopher R., Sterling, Teague, Clark, Wyatt T., Ru, Yuanbin, Trinidad, Marena, Nguyen, Hong Phuc, Luu, Amanda R., Wendt, Daniel J., Yogalingam, Gouri, Yu, Guoying Karen, LeBowitz, Jonathan H., Cardon, Lon R.
الحاوية / القاعدة Nat Commun (2021)

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

بواسطة 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
الحاوية / القاعدة J Biol Chem (2020)

Correction: Intermittent enzyme replacement therapy prevents Neu1 deficiency

بواسطة 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
الحاوية / القاعدة J Biol Chem (2020)

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

بواسطة 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
الحاوية / القاعدة J Biol Chem (2020)

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

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

Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatm...

بواسطة 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
الحاوية / القاعدة Drug Deliv Transl Res (2020)

Assessment of predicted enzymatic activity of alpha-N-acetylglucosaminidase (NAGLU) variants of unknown significance for CAGI 2016

بواسطة Clark, Wyatt T., Kasak, Laura, Bakolitsa, Constantina, Hu, Zhiqiang, Andreoletti, Gaia, Babbi, Giulia, Bromberg, Yana, Casadio, Rita, Dunbrack, Roland, Folkman, Lukas, Ford, Colby T., Jones, David, Katsonis, Panagiotis, Kundu, Kunal, Lichtarge, Olivier, Martelli, Pier Luigi, Mooney, Sean D., Nodzak, Conor, Pal, Lipika R., Radivojac, Predrag, Savojardo, Castrense, Shi, Xinghua, Zhou, Yaoqi, Uppal, Aneeta, Xu, Qifang, Yin, Yizhou, Pejaver, Vikas, Wang, Meng, Wei, Liping, Moult, John, Yu, G. Karen, Brenner, Steven E., LeBowitz, Jonathan H.
الحاوية / القاعدة Hum Mutat (2019)

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