Nietupski JB - Search Results

1

Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease.

Ashe KM, Budman E, Bangari DS, Siegel CS, Nietupski JB, Wang B, Desnick RJ, Scheule RK, Leonard JP, Cheng SH, Marshall J. Ashe KM, et al. Among authors: nietupski jb. Mol Med. 2015 Apr 30;21(1):389-99. doi: 10.2119/molmed.2015.00088. Mol Med. 2015. PMID: 25938659 Free PMC article.

2

Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.

Ashe KM, Bangari D, Li L, Cabrera-Salazar MA, Bercury SD, Nietupski JB, Cooper CG, Aerts JM, Lee ER, Copeland DP, Cheng SH, Scheule RK, Marshall J. Ashe KM, et al. Among authors: nietupski jb. PLoS One. 2011;6(6):e21758. doi: 10.1371/journal.pone.0021758. Epub 2011 Jun 29. PLoS One. 2011. PMID: 21738789 Free PMC article.

3

Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.

Nietupski JB, Pacheco JJ, Chuang WL, Maratea K, Li L, Foley J, Ashe KM, Cooper CG, Aerts JM, Copeland DP, Scheule RK, Cheng SH, Marshall J. Nietupski JB, et al. Mol Genet Metab. 2012 Apr;105(4):621-8. doi: 10.1016/j.ymgme.2012.01.020. Epub 2012 Feb 1. Mol Genet Metab. 2012. PMID: 22366055

4

CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.

Marshall J, Sun Y, Bangari DS, Budman E, Park H, Nietupski JB, Allaire A, Cromwell MA, Wang B, Grabowski GA, Leonard JP, Cheng SH. Marshall J, et al. Among authors: nietupski jb. Mol Ther. 2016 Jun;24(6):1019-1029. doi: 10.1038/mt.2016.53. Epub 2016 Mar 7. Mol Ther. 2016. PMID: 26948439 Free PMC article.

5

Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity.

Marshall J, Nietupski JB, Park H, Cao J, Bangari DS, Silvescu C, Wilper T, Randall K, Tietz D, Wang B, Ying X, Leonard JP, Cheng SH. Marshall J, et al. Among authors: nietupski jb. Mol Ther. 2019 Aug 7;27(8):1495-1506. doi: 10.1016/j.ymthe.2019.05.018. Epub 2019 Jun 4. Mol Ther. 2019. PMID: 31208914 Free PMC article.

6

Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression.

Nietupski JB, Hurlbut GD, Ziegler RJ, Chu Q, Hodges BL, Ashe KM, Bree M, Cheng SH, Gregory RJ, Marshall J, Scheule RK. Nietupski JB, et al. Mol Ther. 2011 Nov;19(11):1999-2011. doi: 10.1038/mt.2011.119. Epub 2011 Jun 28. Mol Ther. 2011. PMID: 21712814 Free PMC article.

7

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

McEachern KA, Nietupski JB, Chuang WL, Armentano D, Johnson J, Hutto E, Grabowski GA, Cheng SH, Marshall J. McEachern KA, et al. Among authors: nietupski jb. J Gene Med. 2006 Jun;8(6):719-29. doi: 10.1002/jgm.901. J Gene Med. 2006. PMID: 16528760

8

Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy.

Hurlbut GD, Ziegler RJ, Nietupski JB, Foley JW, Woodworth LA, Meyers E, Bercury SD, Pande NN, Souza DW, Bree MP, Lukason MJ, Marshall J, Cheng SH, Scheule RK. Hurlbut GD, et al. Among authors: nietupski jb. Mol Ther. 2010 Nov;18(11):1983-94. doi: 10.1038/mt.2010.175. Epub 2010 Aug 24. Mol Ther. 2010. PMID: 20736932 Free PMC article.

9

Cationic lipid structure and formulation considerations for optimal gene transfection of the lung.

Marshall J, Nietupski JB, Lee ER, Siegel CS, Rafter PW, Rudginsky SA, Chang CD, Eastman SJ, Harris DJ, Scheule RK, Cheng SH. Marshall J, et al. Among authors: nietupski jb. J Drug Target. 2000;7(6):453-69. doi: 10.3109/10611860009102219. J Drug Target. 2000. PMID: 10758915

10

Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model.

Marshall J, McEachern KA, Kyros JA, Nietupski JB, Budzinski T, Ziegler RJ, Yew NS, Sullivan J, Scaria A, van Rooijen N, Barranger JA, Cheng SH. Marshall J, et al. Among authors: nietupski jb. Mol Ther. 2002 Aug;6(2):179-89. doi: 10.1006/mthe.2002.0650. Mol Ther. 2002. PMID: 12161184 Free article.

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