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Brain : a Journal of Neurology|November 30, 2017
Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapyRebecca J Holley, Stuart M Ellison, Daniel Fil, et al.
American Journal of Human Genetics|January 10, 2002
Genomic screening of fibroblast growth-factor receptor 2 reveals a wide spectrum of mutations in patients with syndromic craniosynostosisShih-hsin Kan, Navaratnam Elanko, David Johnson, et al.
Comparative Medicine|April 16, 2013
Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type ICharles H Vite, Igor Nestrasil, Anton Mlikotic, et al.
Experimental Neurology|July 30, 2015
Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis IJames M Provenzale, Igor Nestrasil, Steven Chen, et al.
Molecular Therapy. Methods & Clinical Development|June 9, 2015
A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type IDaniel C Mendez, Alexander E Stover, Anthony D Rangel, et al.
Molecular Genetics and Metabolism|July 27, 2010
Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type IPatricia I Dickson, Stephen Hanson, Michael F McEntee, et al.
The Journal of Biological Chemistry|June 24, 2022
Impaired mitophagy in Sanfilippo a mice causes hypertriglyceridemia and brown adipose tissue activationMiguel Tillo, William C Lamanna, Chrissa A Dwyer, et al.
Proceedings of the National Academy of Sciences of the United States of America|October 1, 2014
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIBShih-Hsin Kan, Mika Aoyagi-Scharber, Steven Q Le, et al.
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