A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease
McEachern, Kerry Anne, Fung, John, Komarnitsky, Svetlana, Siegel, Craig S., Chuang, Wei-Lien, Hutto, Elizabeth, Shayman, James A., Grabowski, Gregory A., Aerts, Johannes M.F.G., Cheng, Seng H., Copeland, Diane P., Marshall, John
Published in Molecular genetics and metabolism (01.07.2007)
Published in Molecular genetics and metabolism (01.07.2007)
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Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
Ashe, Karen M, Budman, Eva, Bangari, Dinesh S, Siegel, Craig S, Nietupski, Jennifer B, Wang, Bing, Desnick, Robert J, Scheule, Ronald K, Leonard, John P, Cheng, Seng H, Marshall, John
Published in Molecular medicine (Cambridge, Mass.) (30.04.2015)
Published in Molecular medicine (Cambridge, Mass.) (30.04.2015)
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Process Development of a GCS Inhibitor Including Demonstration of Lossen Rearrangement on Kilogram Scale
Zhao, Jin, Gimi, Rayomand, Katti, Sanjeev, Reardon, Michael, Nivorozhkin, Vitaly, Konowicz, Paul, Lee, Edward, Sole, Lynne, Green, Jerome, Siegel, Craig S
Published in Organic process research & development (15.05.2015)
Published in Organic process research & development (15.05.2015)
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Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy
Marshall, John, McEachern, Kerry Anne, Chuang, Wei-Lien, Hutto, Elizabeth, Siegel, Craig S, Shayman, James A, Grabowski, Greg A, Scheule, Ronald K, Copeland, Diane P, Cheng, Seng H
Published in Journal of inherited metabolic disease (01.06.2010)
Published in Journal of inherited metabolic disease (01.06.2010)
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Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung
Lee, E R, Marshall, J, Siegel, C S, Jiang, C, Yew, N S, Nichols, M R, Nietupski, J B, Ziegler, R J, Lane, M B, Wang, K X, Wan, N C, Scheule, R K, Harris, D J, Smith, A E, Cheng, S H
Published in Human gene therapy (10.09.1996)
Published in Human gene therapy (10.09.1996)
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Optimization of formulations and conditions for the aerosol delivery of functional cationic lipid:DNA complexes
Eastman, S J, Tousignant, J D, Lukason, M J, Murray, H, Siegel, C S, Constantino, P, Harris, D J, Cheng, S H, Scheule, R K
Published in Human gene therapy (10.02.1997)
Published in Human gene therapy (10.02.1997)
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Scalable Syntheses of Chirally Pure Mono-protected and Orthogonally Di-protected cis-3,4-Diaminotetrahydrofurans
Weiberth, Franz J., Bourque, Andre J., Elenbaas, Steven, Hanna, Reda, Powers, Matthew R., Siegel, Craig S., Zhao, Jin
Published in Synthesis (Stuttgart) (18.09.2017)
Published in Synthesis (Stuttgart) (18.09.2017)
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Elovl1 inhibition reduced very long chain fatty acids in a mouse model of adrenoleukodystrophy
Huang, Jeremy, Freed, Brian, Hanus, Martin, Ming Sum Ruby Chiang, Brezzani, Alexander, Luo, Yongyi, Li, Yihang, Lam, Becky, Holley, Stephanie, Gans, Joseph, Dostalova, Zuzana, Tang, Buyun, Phaneuf, Clifford, Teeple, Erin, Parisi, Laura, Guo, Lilu, Zhao, Zhonglin, Kinton, Sofia N, Dwyer, Jacquelyn, Teixeira, Sandrine, Ma, Hong, Asmussen, Gary, Mclaren, Rajashree, Wang, Donghui, Baker, Ann, Siegel, Craig S, Fink, David, Randall, Kristen, Belenky, Alexei, Venugopal, Suchitra, Gaglia, Giorgio, Johnson, Jennifer, Bangari, Dinesh S, Sergio Pablo Sardi, Zhang, Bailin, Alexander, Michel, Proto, Jonathan D, Kloss, Alla, Gladysheva, Tatiana, Kayatekin, Can
Published in bioRxiv (11.11.2024)
Published in bioRxiv (11.11.2024)
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Cationic lipid structure and formulation considerations for optimal gene transfection of the lung
Marshall, J, Nietupski, J B, Lee, E R, Siegel, C S, Rafter, P W, Rudginsky, S A, Chang, C D, Eastman, S J, Harris, D J, Scheule, R K, Cheng, S H
Published in Journal of drug targeting (2000)
Published in Journal of drug targeting (2000)
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