Research output per year
Research output per year
Personal profile
Research interests
My laboratory studies the pathogenic mechanisms leading to the clinical signs associated with lysosomal storage diseases. We also develop effective therapeutic approaches for this class of inherited pediatric disease. Specifically, we have developed recombinant enzyme replacement, stem cell-mediated and gene transfer therapies in our mouse models of disease.
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Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome
Kan, S. H., Elsharkawi, I., Le, S. Q., Prill, H., Mangini, L., Cooper, J. D., Lawrence, R., Sands, M. S., Crawford, B. E. & Dickson, P. I., Jun 2021, In: Molecular genetics and metabolism. 133, 2, p. 185-192 8 p.Research output: Contribution to journal › Article › peer-review
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Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease
Li, Y., Miller, C. A., Shea, L. K., Jiang, X., Guzman, M. A., Chandler, R. J., Ramakrishnan, S. M., Smith, S. N., Venditti, C. P., Vogler, C. A., Ory, D. S., Ley, T. J. & Sands, M. S., Feb 3 2021, In: Molecular Therapy. 29, 2, p. 691-701 11 p.Research output: Contribution to journal › Article › peer-review
3 Scopus citations -
Krabbe disease: New hope for an old disease
Bradbury, A. M., Bongarzone, E. R. & Sands, M. S., May 1 2021, In: Neuroscience Letters. 752, 135841.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
Spinal manifestations of CLN1 disease start during the early postnatal period
Nelvagal, H. R., Dearborn, J. T., Ostergaard, J. R., Sands, M. S. & Cooper, J. D., Feb 2021, In: Neuropathology and Applied Neurobiology. 47, 2, p. 251-267 17 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease
Heller, G. J., Marshall, M. S., Issa, Y., Marshall, J. N., Nguyen, D., Rue, E., Pathmasiri, K. C., Domowicz, M. S., van Breemen, R. B., Tai, L. M., Cologna, S. M., Crocker, S. J., Givogri, M. I., Sands, M. S. & Bongarzone, E. R., May 5 2021, In: Molecular Therapy. 29, 5, p. 1883-1902 20 p.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations