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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Research Output
Cell-autonomous expression of the acid hydrolase galactocerebrosidase
Mikulka, C. R., Dearborn, J. T., Benitez, B. A., Strickland, A., Liu, L., Milbrandt, J. & Sands, M. S., Apr 21 2020, In : Proceedings of the National Academy of Sciences of the United States of America. 117, 16, p. 9032-9041 10 p.Research output: Contribution to journal › Article
Cell-Type-Specific Profiling of Alternative Translation Identifies Regulated Protein Isoform Variation in the Mouse Brain
Sapkota, D., Lake, A. M., Yang, W., Yang, C., Wesseling, H., Guise, A., Uncu, C., Dalal, J. S., Kraft, A. W., Lee, J. M., Sands, M. S., Steen, J. A. & Dougherty, J. D., Jan 15 2019, In : Cell Reports. 26, 3, p. 594-607.e7Research output: Contribution to journal › Article
Open Access
8
Scopus
citations
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target
Li, Y., Xu, Y., Benitez, B. A., Nagree, M. S., Dearborn, J. T., Jiang, X., Guzman, M. A., Woloszynek, J. C., Giaramita, A., Yip, B. K., Elsbernd, J., Babcock, M. C., Lo, M., Fowler, S. C., Wozniak, D. F., Vogler, C. A., Medin, J. A., Crawford, B. E. & Sands, M. S., Oct 1 2019, In : Proceedings of the National Academy of Sciences of the United States of America. 116, 40, p. 20097-20103 7 p.Research output: Contribution to journal › Article
Open Access
7
Scopus
citations
A HILIC-MS/MS method for simultaneous quantification of the lysosomal disease markers galactosylsphingosine and glucosylsphingosine in mouse serum
Sidhu, R., Mikulka, C. R., Fujiwara, H., Sands, M. S., Schaffer, J. E., Ory, D. S. & Jiang, X., Jul 2018, In : Biomedical Chromatography. 32, 7, e4235.Research output: Contribution to journal › Article
6
Scopus
citations
A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I
Le, S. Q., Kan, S. H., Clarke, D., Sanghez, V., Egeland, M., Vondrak, K. N., Doherty, T. M., Vera, M. U., Iacovino, M., Cooper, J. D., Sands, M. S. & Dickson, P. I., Mar 16 2018, In : Molecular Therapy - Methods and Clinical Development. 8, p. 42-51 10 p.Research output: Contribution to journal › Article
Open Access
7
Scopus
citations