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Current Opinion in Molecular Therapeutics|September 12, 2002
Gene therapy for the lysosomal storage disordersMario A Cabrera-Salazar, Enrico Novelli, John A Barranger
The Journal of Pediatrics|July 20, 2005
Radiological evidence of early cerebral microvascular disease in young children with Fabry diseaseMario A Cabrera-Salazar, Erin O'Rourke, Gustavo Charria-Ortiz, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry|May 20, 2004
Correlation of surrogate markers of Gaucher disease. Implications for long-term follow up of enzyme replacement therapyMario A Cabrera-Salazar, Erin O'Rourke, Nadene Henderson, 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.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 26, 2012
Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type AJie Bu, Karen M Ashe, John Bringas, 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.
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