AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency by Lei Zhu, Brandon Tan, Selina S. Dwight, Brendan Beahm, Matt Wilsey, Brett E. Crawford, Becky Schweighardt, Jennifer W. Cook, Thomas Wechsler, William F. Mueller

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Natural history study of glycan accumulation in large animal models of GM2 gangliosidoses. by Catlyn Cavender, Linley Mangini, Jeremy L Van Vleet, Carley Corado, Emma McCullagh, Heather L Gray-Edwards, Douglas R Martin, Brett E Crawford, Roger Lawrence

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Characterization of glycan substrates accumulating in GM1 Gangliosidosis by Roger Lawrence, Jeremy L. Van Vleet, Linley Mangini, Adam Harris, Nathan Martin, Wyatt Clark, Sanjay Chandriani, Jonathan H. LeBowitz, Roberto Giugliani, Alessandra d'Azzo, Gouri Yogalingam, Brett E. Crawford

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Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease. by Marcelina Malinowska, Fiona L Wilkinson, Kia J Langford-Smith, Alex Langford-Smith, Jillian R Brown, Brett E Crawford, Marie T Vanier, Grzegorz Grynkiewicz, Rob F Wynn, J Ed Wraith, Grzegorz Wegrzyn, Brian W Bigger

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Central nervous system pathology in preclinical MPS IIIB dogs reveals progressive changes in clinically relevant brain regions by Martin T. Egeland, Marta M. Tarczyluk-Wells, Melissa M. Asmar, Evan G. Adintori, Roger Lawrence, Elizabeth M. Snella, Jackie K. Jens, Brett E. Crawford, Jill C. M. Wait, Emma McCullagh, Jason Pinkstaff, Jonathan D. Cooper, N. Matthew Ellinwood

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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice by Mika Aoyagi-Scharber, Danielle Crippen-Harmon, Roger Lawrence, Jon Vincelette, Gouri Yogalingam, Heather Prill, Bryan K. Yip, Brian Baridon, Catherine Vitelli, Amanda Lee, Olivia Gorostiza, Evan G. Adintori, Wesley C. Minto, Jeremy L. Van Vleet, Bridget Yates, Sara Rigney, Terri M. Christianson, Pascale M.N. Tiger, Melanie J. Lo, John Holtzinger, Paul A. Fitzpatrick, Jonathan H. LeBowitz, Sherry Bullens, Brett E. Crawford, Stuart Bunting

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