Terri Christianson

Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

Shih‐hsin Kan, Mika Aoyagi-Scharber, Steven Q. Le et al. · Proceedings of the National Academy of Sciences · 2014 · 117 citations · Full text

Pharmacy, Biochemistry, Molecular Medicine +15

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

S. Rosén, Stefan Tiefenbacher, Mary Robinson et al. · Blood · 2020 · 71 citations · Full text

Life Sciences, Genetics, Molecular Biology +13

Enzyme Replacement in a Human Model of Mucopolysaccharidosis IVA In Vitro and Its Biodistribution in the Cartilage of Wild Type Mice

Melita Dvorak‐Ewell, Daniel Wendt, Chuck Hague et al. · PLoS ONE · 2010 · 61 citations · Full text

Life Sciences, Biochemistry, Molecular Medicine +15

Lipoprotein Receptor Binding, Cellular Uptake, and Lysosomal Delivery of Fusions between the Receptor-associated Protein (RAP) and α-l-Iduronidase or Acid α-Glucosidase

William S. Prince, Lynn M. McCormick, Dan J. Wendt et al. · Journal of Biological Chemistry · 2004 · 47 citations · Full text

Life Sciences, Cell Biology, Molecular Biology +17

Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice

Joseph C. Chen, Amanda Luu, Nathan Wise et al. · Journal of Biological Chemistry · 2019 · 40 citations · Full text

Life Sciences, Biochemistry, Neurobiology +17