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Journal of Molecular Medicine (Berlin, Germany)|July 7, 2012
Rapid identification of novel functional promoters for gene therapyIan A Pringle, Deborah R Gill, Mary M Connolly, et al.
Experimental Neurology|July 23, 2011
Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusionRobin J Ziegler, Ernesto A Salegio, James C Dodge, et al.
Plos One|August 23, 2012
Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher diseaseMario A Cabrera-Salazar, Matthew Deriso, Scott D Bercury, et al.
Acta Biochimica Et Biophysica Sinica|January 1, 2010
Low-frequency ultrasound increases non-viral gene transfer to the mouse lungStefania Xenariou, Hai-Dong Liang, Uta Griesenbach, et al.
Molecular Genetics and Metabolism|February 28, 2012
Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C miceJennifer B Nietupski, Joshua J Pacheco, Wei-Lien Chuang, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 20, 2007
Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten diseaseMario A Cabrera-Salazar, Eric M Roskelley, Jie Bu, et al.
Plos One|July 9, 2011
Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff diseaseKaren M Ashe, Dinesh Bangari, Lingyun Li, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 26, 2010
Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapyGregory D Hurlbut, Robin J Ziegler, Jennifer B Nietupski, et al.
Molecular Genetics and Metabolism|June 18, 2010
Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe diseaseKaren M Ashe, Kristin M Taylor, Qiuming Chu, et al.
The Journal of Gene Medicine|November 26, 2009
Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavityEmma Holder, Barbara Stevenson, Raymond Farley, et al.
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