Karen M. Ashe

Medicine · Health Sciences

8h-index475citations13works0.02yr avg
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Research Topics

Lysosomal Storage Disorders Research(12), Trypanosoma species research and implications(5), Carbohydrate Chemistry and Synthesis(4), Virus-based gene therapy research(2), Cellular transport and secretion(2)

Publications13 total

Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
Molecular Medicine·2015· 99 citedOpen Access
α-Galactosidase A Knockout Mice
American Journal Of Pathology·2014· 41 citedOpen Access
A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease
Molecular Genetics and Metabolism·2013
Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
Molecular Therapy·2012· 29 citedOpen Access
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
Substrate Reduction Augments the Efficacy of Enzyme Therapy in a Mouse Model of Fabry Disease
PLoS ONE·2010· 72 citedOpen Access
Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease
Molecular Genetics and Metabolism·2010· 46 cited
90. Efficacy of Genz-529468-mediated inhibition of glucosylceramide synthase in a mouse model of Sandhoff disease
Molecular Genetics and Metabolism·2010· 1 cited
91. Management of lysosomal glycosphingolipid levels in animal models of Gaucher and Fabry disease with enzyme and substrate reduction therapies
Molecular Genetics and Metabolism·2010
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

Frequent Co-authors

Seng H. Cheng(13), John Marshall(12), Ronald K. Scheule(11), Jennifer B. Nietupski(8), Dinesh S. Bangari(6), Robert J. Desnick(5), Diane Copeland(5), John P. Leonard(4), Wei‐Lien Chuang(4), Eva Budman(3), Christopher G. F. Cooper(3), Johannes M. F. G. Aerts(3), Qiuming Chu(3), Bing Wang(2), Joshua Pacheco(2), Lingyun Li(2), Mario A. Cabrera-Salazar(2), Robin J. Ziegler(2), Bradley L. Hodges(2), Kristin M. Taylor(2)