Jennifer B. Nietupski

Sanofi (United States) — United States

Medicine · Health Sciences

15h-index2.0kcitations27works0.02yr avg
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Research Topics

Lysosomal Storage Disorders Research(14), Virus-based gene therapy research(9), RNA Interference and Gene Delivery(8), Carbohydrate Chemistry and Synthesis(5), Cellular transport and secretion(4)

Publications27 total

Ultrasound imaging for assessing aortic phenotypes: A preclinical tool for measuring cardiac disease model progression and therapeutic effect
WFUMB Ultrasound Open·2024Open Access
Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity
Molecular Therapy·2019· 51 citedOpen Access
Publisher Correction: Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1
Scientific Reports·2018Open Access
Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1
Scientific Reports·2018· 4 citedOpen Access
Efficacy of Genz-682452-mediated inhibition of glucosylceramide synthase in a mouse model of Sandhoff disease
Molecular Genetics and Metabolism·2018
CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease
Molecular Therapy·2016· 100 citedOpen Access
Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
Molecular Medicine·2015· 99 citedOpen Access
A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease
Molecular Genetics and Metabolism·2013
Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice
Molecular Genetics and Metabolism·2012· 61 cited
Glucosylceramide Synthase Inhibition Reduces Gb3 and Lyso-Gb3 in a Mouse Model Of Fabry Disease
Molecular Genetics and Metabolism·2012
Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease
PLoS ONE·2011· 72 citedOpen Access
Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression
Molecular Therapy·2011· 54 citedOpen Access
Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
Molecular Therapy·2010· 134 citedOpen Access
90. Efficacy of Genz-529468-mediated inhibition of glucosylceramide synthase in a mouse model of Sandhoff disease
Molecular Genetics and Metabolism·2010· 1 cited
AAV8‐mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease
The Journal of Gene Medicine·2006· 66 cited
217. Comparison of Hepatic Vein and Peripheral Vein Delivery of an AAV2/8 Vector Encoding Human a-Galactosidase A to Rhesus Macaques
Molecular Therapy·2006Open Access
Demonstration of Feasibility of In Vivo Gene Therapy for Gaucher Disease Using a Chemically Induced Mouse Model
Molecular Therapy·2002· 53 citedOpen Access
Comprehensive Analysis of the Acute Toxicities Induced by Systemic Administration of Cationic Lipid:Plasmid DNA Complexes in Mice
Human Gene Therapy·2000· 233 cited
Cationic Lipid Structure and Formulation Considerations for Optimal Gene Transfection of the Lung
Journal of drug targeting·2000· 38 cited
Restoration of Cyclic Adenosine Monophosphate–Stimulated Chloride Channel Activity in Human Cystic Fibrosis Tracheobronchial Submucosal Gland Cells by Adenovirus-Mediated and Cationic Lipid–Mediated Gene Transfer
American Journal of Respiratory Cell and Molecular Biology·1999· 7 cited
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Frequent Co-authors

Seng H. Cheng(17), John Marshall(16), Ronald K. Scheule(15), Dinesh S. Bangari(8), Karen M. Ashe(8), John P. Leonard(7), Robin J. Ziegler(5), Craig Siegel(4), Bing Wang(4), Drew Tietz(4), Edward R. Lee(3), Nelson S. Yew(3), David J. Harris(3), Eva Budman(3), Hye Jung Park(3), Robert J. Desnick(3), Johannes M. F. G. Aerts(3), Diane Copeland(3), Wei‐Lien Chuang(3), Canwen Jiang(2)