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Select item 105509791.

Disease pathology signatures in a mouse model of Mucopolysaccharidosis type IIIB

Ralitsa Petrova, Abhijeet R. Patil, Vivian Trinh, Kathryn E. McElroy, Minoti Bhakta, Jason Tien, David S. Wilson, Liling Warren, Jennifer R. Stratton

Sci Rep. 2023; 13: 16699. Published online 2023 Oct 4. doi: 10.1038/s41598-023-42431-4

PMCID:: PMC10550979

Article PDF–3.6M

Select item 98517422.

Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector

Courtney J Rouse, Kimberley Hawkins, Nadia Kabbej, Justin Dalugdug, Aishwarya Kunta, Mi-Jung Kim, Shinichi Someya, Zachary Herbst, Michael Gelb, Isabella Dinelli, Elizabeth Butterworth, Darin J Falk, Erinn Rosenkrantz, Hamza Elmohd, Hamid Khaledi, Samar Mowafy, Frederick Ashby, Coy D Heldermon

Hum Mol Genet. 2023 Feb 1; 32(3): 417–430. Published online 2022 Aug 23. doi: 10.1093/hmg/ddac209

PMCID:: PMC9851742

Article PDF–1.7M

Select item 67301923.

Improved Behavior and Neuropathology in the Mouse Model of Sanfilippo Type IIIB Disease after Adeno-Associated Virus-Mediated Gene Transfer in the Striatum

Arnaud Cressant, Nathalie Desmaris, Lucie Verot, Thomas Bréjot, Roseline Froissart, Marie-T. Vanier, Irène Maire, Jean Michel Heard

J Neurosci. 2004 Nov 10; 24(45): 10229–10239. doi: 10.1523/JNEUROSCI.3558-04.2004

PMCID:: PMC6730192

Article PDF–789K

Select item 61037064.

Trehalose reduces retinal degeneration, neuroinflammation and storage burden caused by a lysosomal hydrolase deficiency

Parisa Lotfi, Dennis Y. Tse, Alberto Di Ronza, Michelle L. Seymour, Giuseppe Martano, Jonathan D. Cooper, Fred A. Pereira, Maria Passafaro, Samuel M. Wu, Marco Sardiello

Autophagy. 2018; 14(8): 1419–1434. Published online 2018 Jul 23. doi: 10.1080/15548627.2018.1474313

PMCID:: PMC6103706

Article PDF–3.0M

Select item 46608515.

Electrophysiological and Histological Characterization of Rod-Cone Retinal Degeneration and Microglia Activation in a Mouse Model of Mucopolysaccharidosis Type IIIB

Dennis Y. Tse, Parisa Lotfi, David L. Simons, Marco Sardiello, Samuel M. Wu

Sci Rep. 2015; 5: 17143. Published online 2015 Nov 26. doi: 10.1038/srep17143

PMCID:: PMC4660851

Article PDF–2.6M

Select item 44930276.

The Murine Model of Mucopolysaccharidosis IIIB Develops Cardiopathies over Time Leading to Heart Failure

Gabriele Giacomo Schiattarella, Giuliana Cerulo, Valeria De Pasquale, Pasquale Cocchiaro, Orlando Paciello, Luigi Avallone, Maria Paola Belfiore, Francesca Iacobellis, Daniele Di Napoli, Fabio Magliulo, Cinzia Perrino, Bruno Trimarco, Giovanni Esposito, Paola Di Natale, Luigi Michele Pavone

PLoS One. 2015; 10(7): e0131662. Published online 2015 Jul 6. doi: 10.1371/journal.pone.0131662

PMCID:: PMC4493027

Article PDF–21M

Select item 42056717.

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, Jon Vincelette, Kazuhiro Ohmi, Sherry Bullens, Daniel J. Wendt, Terri M. Christianson, Pascale M. N. Tiger, Jillian R. Brown, Roger Lawrence, Bryan K. Yip, John Holtzinger, Anil Bagri, Danielle Crippen-Harmon, Kristen N. Vondrak, Zhi Chen, Chuck M. Hague, Josh C. Woloszynek, Diana S. Cheung, Katherine A. Webster, Evan G. Adintori, Melanie J. Lo, Wesley Wong, Paul A. Fitzpatrick, Jonathan H. LeBowitz, Brett E. Crawford, Stuart Bunting, Patricia I. Dickson, Elizabeth F. Neufeld

Proc Natl Acad Sci U S A. 2014 Oct 14; 111(41): 14870–14875. Published online 2014 Sep 29. doi: 10.1073/pnas.1416660111

PMCID:: PMC4205671

Article PDF–1.1M

Select item 38369788.

Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood

Bartholomew J. Naughton, F. Jason Duncan, Darren Murrey, Tierra Ware, Aaron Meadows, Douglas M. McCarty, Haiyan Fu

PLoS One. 2013; 8(11): e80142. Published online 2013 Nov 21. doi: 10.1371/journal.pone.0080142

PMCID:: PMC3836978

Article PDF–12M

Select item 34579359.

Peripheral Nervous System Neuropathology and Progressive Sensory Impairments in a Mouse Model of Mucopolysaccharidosis IIIB

Haiyan Fu, Julianne D. Bartz, Robert L. Stephens, Jr, Douglas M. McCarty

PLoS One. 2012; 7(9): e45992. Published online 2012 Sep 25. doi: 10.1371/journal.pone.0045992

PMCID:: PMC3457935

Article PDF–3.7M

Select item 304976410.

Blood-Brain Barrier Impairment in an Animal Model of MPS III B

Svitlana Garbuzova-Davis, Michael K. Louis, Edward M. Haller, Hiranya M. Derasari, Ashley E. Rawls, Paul R. Sanberg

PLoS One. 2011; 6(3): e16601. Published online 2011 Mar 7. doi: 10.1371/journal.pone.0016601

PMCID:: PMC3049764

Article PDF–2.6M

Select item 299573611.

Genistein Improves Neuropathology and Corrects Behaviour in a Mouse Model of Neurodegenerative Metabolic Disease

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

PLoS One. 2010; 5(12): e14192. Published online 2010 Dec 1. doi: 10.1371/journal.pone.0014192

PMCID:: PMC2995736

Article PDF–4.3M

Select item 299328012.

Storage Vesicles in Neurons Are Related to Golgi Complex Alterations in Mucopolysaccharidosis IIIB

Sandrine Vitry, Julie Bruyère, Michaël Hocquemiller, Stéphanie Bigou, Jérôme Ausseil, Marie-Anne Colle, Marie-Christine Prévost, Jean Michel Heard

Am J Pathol. 2010 Dec; 177(6): 2984–2999. doi: 10.2353/ajpath.2010.100447

PMCID:: PMC2993280

Article PDF–2.2M

Select item 113897213.

Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector

Paola Di Natale, Carmela Di Domenico, Nadia Gargiulo, Sigismondo Castaldo, Enrico Gonzalez Y Reyero, Pratibha Mithbaokar, Mario De Felice, Antonia Follenzi, Luigi Naldini, Guglielmo R. D. Villani

Biochem J. 2005 Jun 1; 388(Pt 2): 639–646. Prepublished online 2005 Jan 13. Published online 2005 May 24. doi: 10.1042/BJ20041702

PMCID:: PMC1138972

Article PDF–402K

Select item 936403514.

Impaired mitophagy in Sanfilippo a mice causes hypertriglyceridemia and brown adipose tissue activation

Miguel Tillo, William C. Lamanna, Chrissa A. Dwyer, Daniel R. Sandoval, Ariane R. Pessentheiner, Norah Al-Azzam, Stéphane Sarrazin, Jon C. Gonzales, Shih-Hsin Kan, Alexander Y. Andreyev, Nicholas Schultheis, Bryan E. Thacker, Charles A. Glass, Patricia I. Dickson, Raymond Y. Wang, Scott B. Selleck, Jeffrey D. Esko, Philip L.S.M. Gordts

J Biol Chem. 2022 Aug; 298(8): 102159. Published online 2022 Jun 22. doi: 10.1016/j.jbc.2022.102159

PMCID:: PMC9364035

Article PDF–3.2M

Select item 819584815.

Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome

Shih-hsin Kan, Ibrahim Elsharkawi, Steven Q. Le, Heather Prill, Linley Mangini, Jonathan D. Cooper, Roger Lawrence, Mark S. Sands, Brett E. Crawford, Patricia I. Dickson

Mol Genet Metab. Author manuscript; available in PMC 2022 Jun 1.

Published in final edited form as: Mol Genet Metab. 2021 Jun; 133(2): 185–192. Published online 2021 Mar 31. doi: 10.1016/j.ymgme.2021.03.013

PMCID:: PMC8195848

Article PDF–1.5M

Select item 570372016.

Deleting the mouse Hsd17b1 gene results in a hypomorphic Naglu allele and a phenotype mimicking a lysosomal storage disease

Heli Jokela, Janne Hakkarainen, Laura Kätkänaho, Pirjo Pakarinen, Suvi T. Ruohonen, Manuel Tena-Sempere, Fu-Ping Zhang, Matti Poutanen

Sci Rep. 2017; 7: 16406. Published online 2017 Nov 27. doi: 10.1038/s41598-017-16618-5

PMCID:: PMC5703720

Article PDF–10M

Select item 194501517.

Development of Sensory, Motor and Behavioral Deficits in the Murine Model of Sanfilippo Syndrome Type B

Coy D. Heldermon, Anne K. Hennig, Kevin K. Ohlemiller, Judith M. Ogilvie, Erik D. Herzog, Annalisa Breidenbach, Carole Vogler, David F. Wozniak, Mark S. Sands

PLoS One. 2007; 2(8): e772. Published online 2007 Aug 22. doi: 10.1371/journal.pone.0000772

PMCID:: PMC1945015

Article PDF–1.2M

Select item 14993118.

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

Kazuhiro Ohmi, David S. Greenberg, Kavitha S. Rajavel, Sergey Ryazantsev, Hong Hua Li, Elizabeth F. Neufeld

Proc Natl Acad Sci U S A. 2003 Feb 18; 100(4): 1902–1907. Published online 2003 Feb 7. doi: 10.1073/pnas.252784899

PMCID:: PMC149931

Article PDF–485K

Select item 723698319.

Aortopathies in mouse models of Pompe, Fabry and Mucopolysaccharidosis IIIB lysosomal storage diseases

Maria Paola Belfiore, Francesca Iacobellis, Emma Acampora, Martina Caiazza, Marta Rubino, Emanuele Monda, Maria Rosaria Magaldi, Antonietta Tarallo, Marcella Sasso, Valeria De Pasquale, Roberto Grassi, Salvatore Cappabianca, Paolo Calabrò, Simona Fecarotta, Salvatore Esposito, Giovanni Esposito, Antonio Pisani, Luigi Michele Pavone, Giancarlo Parenti, Giuseppe Limongelli

PLoS One. 2020; 15(5): e0233050. Published online 2020 May 19. doi: 10.1371/journal.pone.0233050

PMCID:: PMC7236983

Article PDF–1.5M

Select item 333878120.

Neuropathology in Mouse Models of Mucopolysaccharidosis Type I, IIIA and IIIB

Fiona L. Wilkinson, Rebecca J. Holley, Kia J. Langford-Smith, Soumya Badrinath, Aiyin Liao, Alex Langford-Smith, Jonathan D. Cooper, Simon A. Jones, J. Ed Wraith, Rob F. Wynn, Catherine L. R. Merry, Brian W. Bigger

PLoS One. 2012; 7(4): e35787. Published online 2012 Apr 27. doi: 10.1371/journal.pone.0035787

PMCID:: PMC3338781

Article PDF–13M

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