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.
Available to Mentor:
- PhD/MSTP Students
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Collaborations and top research areas from the last five years
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Embryonic vitamin D deficiency programs hematopoietic stem cells to induce type 2 diabetes
Oh, J., Riek, A. E., Bauerle, K. T., Dusso, A., McNerney, K. P., Barve, R. A., Darwech, I., Sprague, J. E., Moynihan, C., Zhang, R. M., Kutz, G., Wang, T., Xing, X., Li, D., Mrad, M., Wigge, N. M., Castelblanco, E., Collin, A., Bambouskova, M. & Head, R. D. & 2 others, , Dec 2023, In: Nature communications. 14, 1, 3278.Research output: Contribution to journal › Article › peer-review
Open Access8 Scopus citations -
Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model
Takahashi, K., Eultgen, E. M., Wang, S. H., Rensing, N. R., Nelvagal, H. R., Dearborn, J. T., Danos, O., Buss, N., Sands, M. S., Wong, M. & Cooper, J. D., Jun 15 2023, In: Journal of Clinical Investigation. 133, 12, e165908.Research output: Contribution to journal › Article › peer-review
Open Access5 Scopus citations -
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., Jan 4 2023, In: Molecular Therapy. 31, 1, p. 7-23 17 p.Research output: Contribution to journal › Review article › peer-review
Open Access7 Scopus citations -
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 Access7 Scopus citations -
Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep
Nelvagal, H. R., Eaton, S. L., Wang, S. H., Eultgen, E. M., Takahashi, K., Le, S. Q., Nesbitt, R., Dearborn, J. T., Siano, N., Puhl, A. C., Dickson, P. I., Thompson, G., Murdoch, F., Brennan, P. M., Gray, M., Greenhalgh, S. N., Tennant, P., Gregson, R., Clutton, E. & Nixon, J. & 10 others, , Oct 17 2022, In: Journal of Clinical Investigation. 132, 20, e163107.Research output: Contribution to journal › Article › peer-review
Open Access3 Scopus citations