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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CRISPR-Cas9 Knock-In of T513M and G41S Mutations in the Murine β–Galactosyl-Ceramidase Gene Re-capitulates Early-Onset and Adult-Onset Forms of Krabbe Disease
Rebiai, R., Rue, E., Zaldua, S., Nguyen, D., Scesa, G., Jastrzebski, M., Foster, R., Wang, B., Jiang, X., Tai, L., Brady, S. T., van Breemen, R., Givogri, M. I., Sands, M. S. & Bongarzone, E. R., May 10 2022, In: Frontiers in Molecular Neuroscience. 15, 896314.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
Effects of chronic cannabidiol in a mouse model of naturally occurring neuroinflammation, neurodegeneration, and spontaneous seizures
Dearborn, J. T., Nelvagal, H. R., Rensing, N. R., Takahashi, K., Hughes, S. M., Wishart, T. M., Cooper, J. D., Wong, M. & Sands, M. S., Dec 2022, In: Scientific reports. 12, 1, 11286.Research output: Contribution to journal › Article › peer-review
Open Access -
Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases
Heller, G., Bradbury, A. M., Sands, M. S. & Bongarzone, E. R., 2022, (Accepted/In press) In: Molecular Therapy.Research output: Contribution to journal › Review article › peer-review
Open Access -
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
1 Scopus citations -
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
Open Access18 Scopus citations