Projects per year
Personal profile
Expertise related to UN Sustainable Development Goals
In 2015, UN member states agreed to 17 global Sustainable Development Goals (SDGs) to end poverty, protect the planet and ensure prosperity for all. This person’s work contributes towards the following SDG(s):
Education/Academic qualification
PhD, Models and Gene Therapy for GM1-Gangliosidosis and Morquio Syndrome Type B, University of Minnesota
Aug 14 2014 → Dec 31 2018
Award Date: Dec 31 2018
BS, University of Minnesota
Jan 1 2009 → May 13 2012
Award Date: May 13 2012
Research Interest Keywords
- lysosomal disease
- gene therapy
- gangliosidosis
- mucopolysaccharidosis
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Projects
- 1 Active
Research output
- 12 Article
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Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system
Kim, S., Przybilla, M. J., Whitley, C. B., Ou, L., Al-Kofahi, M. & Jarnes, J. R., Dec 2022, In: Molecular Genetics and Metabolism Reports. 33, 100917.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis
Przybilla, M. J., Stewart, C., Carlson, T. W., Ou, L., Koniar, B. L., Sidhu, R., Kell, P. J., Jiang, X., Jarnes, J. R., O'Sullivan, M. G. & Whitley, C. B., Jun 2021, In: Molecular Genetics and Metabolism Reports. 27, 100748.Research output: Contribution to journal › Article › peer-review
Open Access6 Scopus citations -
A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I
Ou, L., Przybilla, M. J., Ahlat, O., Kim, S., Overn, P., Jarnes, J., O'Sullivan, M. G. & Whitley, C. B., Jun 3 2020, In: Molecular Therapy. 28, 6, p. 1442-1454 13 p.Research output: Contribution to journal › Article › peer-review
Open Access35 Scopus citations -
A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases
Ou, L., Przybilla, M. J., Tăbăran, A. F., Overn, P., O’Sullivan, M. G., Jiang, X., Sidhu, R., Kell, P. J., Ory, D. S. & Whitley, C. B., May 1 2020, In: Gene therapy. 27, 5, p. 226-236 11 p.Research output: Contribution to journal › Article › peer-review
Open Access28 Scopus citations -
Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type B
Przybilla, M. J., Ou, L., Tăbăran, A. F., Jiang, X., Sidhu, R., Kell, P. J., Ory, D. S., O'Sullivan, M. G. & Whitley, C. B., Feb 2019, In: Molecular Genetics and Metabolism. 126, 2, p. 139-150 12 p.Research output: Contribution to journal › Article › peer-review
Open Access17 Scopus citations