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Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency.

E H Birkenmeier, … , L M Maltais, C J Wawrzyniak

E H Birkenmeier, … , L M Maltais, C J Wawrzyniak

Published April 1, 1989
Citation Information: J Clin Invest. 1989;83(4):1258-1266. https://doi.org/10.1172/JCI114010.

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Citations to this article (239)

Title and authors	Publication	Year
Haploinsufficiency of lysosomal enzyme genes in Alzheimer’s disease Benitez BA, Wallace CE, Patel M, Nykanen NP, Yuede CM, Eaton SL, Pottier C, Cetin A, Johnson M, Bevan MT, Gardiner WD, Edwards HM, Doherty BM, Harrigan RT, Kurian D, Wishart TM, Smith C, Cirrito JR, Sands MS	bioRxiv	2024
POLYMER-BASED DRUG DELIVERY SYSTEMS UNDER INVESTIGATION FOR ENZYME REPLACEMENT AND OTHER THERAPIES OF LYSOSOMAL STORAGE DISORDERS Placci M, Giannotti MI, Muro S	Advanced Drug Delivery Reviews	2023
Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome). Poswar FO, Henriques Nehm J, Kubaski F, Poletto E, Giugliani R	Therapeutics and Clinical Risk Management	2022
The beta-glucuronidase intracisternal A particle insertion model results in similar overall MPSVII phenotype as the single base deletion model when on the same C57BL/6J mouse background SC Devanney, JM Gibney, CG Prell, TJ Wronski, JI Aguirre, I Mcdoom, CD Heldermon	Molecular Genetics and Metabolism Reports	2021
Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with mucopolysaccharidosis type VII QH Nguyen, RG Witt, B Wang, C Eikani, J Shea, LK Smith, G Boyle, J Cadaoas, R Sper, JD MacKenzie, S Villeda, TC MacKenzie	Science Translational Medicine	2020
Failures of Endochondral Ossification in the Mucopolysaccharidoses Z Jiang, S Byers, ML Casal, LJ Smith	Current Osteoporosis Reports	2020
Liver background uptake of [18F]FLT in PET imaging Olga Sergeeva, Yifan Zhang, Jonathan Kenyon, Galen Miller-Atkins, Maxim Sergeev, Emily Verbus, Renuka Iyer, Sandra Sexton, Vladimir Kepe, Norbert Avril, Yogen Saunthararajah, Ernst Ricky Chan, Zhenghong Lee	American journal of nuclear medicine and molecular imaging	2020
Mouse methods and models for studies in hearing KK Ohlemiller	The Journal of the Acoustical Society of America	2019
Beta-Glucuronidase Catalyzes Deconjugation and Activation of Curcumin-Glucuronide in Bone AG Kunihiro, PB Luis, JA Brickey, JB Frye, HH Chow, C Schneider, JL Funk	Journal of Natural Products	2019
Delayed development of ossification centers in the tibia of prenatal and early postnatal MPS VII mice Z Jiang, AL Derrick-Roberts, MR Jackson, C Rossouw, CE Pyragius, C Xian, J Fletcher, S Byers	Molecular Genetics and Metabolism	2018
Neuromuscular degeneration and locomotor deficit in a Drosophila model of mucopolysaccharidosis VII is attenuated by treatment with resveratrol S Bar, M Prasad, R Datta	Disease models & mechanisms	2018
rAAV integration and genotoxicity: insights from animal models RJ Chandler, M Sands, CP Venditti	Human Gene Therapy	2017
C-Type Lectin Receptor Dectin-2 Binds to an Endogenous Protein β-Glucuronidase on Dendritic Cells D Mori, K Shibata, S Yamasaki, Y Hoshino	PloS one	2017
Pathogenesis and treatment of spine disease in the mucopolysaccharidoses SH Peck, ML Casal, NR Malhotra, C Ficicioglu, LJ Smith	Molecular Genetics and Metabolism	2016
Clinical course of sly syndrome (mucopolysaccharidosis type VII) AM Montaño, N Lock-Hock, RD Steiner, BH Graham, M Szlago, R Greenstein, M Pineda, A Gonzalez-Meneses, M Çoker, D Bartholomew, MS Sands, R Wang, R Giugliani, A Macaya, G Pastores, AK Ketko, F Ezgü, A Tanaka, L Arash, M Beck, RE Falk, K Bhattacharya, J Franco, KK White, GA Mitchell, L Cimbalistiene, M Holtz, WS Sly	Journal of medical genetics	2016
Integrated analysis of proteome and transcriptome changes in the mucopolysaccharidosis type VII mouse hippocampus MK Parente, R Rozen, SH Seeholzer, JH Wolfe	Molecular Genetics and Metabolism	2016
Reversal of established bone pathology in MPS VII mice following lentiviral-mediated gene therapy AL Derrick-Roberts, K Panir, CE Pyragius, KH Zarrinkalam, GJ Atkins, S Byers	Molecular Genetics and Metabolism	2016
Application of Mouse Models to Research in Hearing and Balance KK Ohlemiller, SM Jones, KR Johnson	Journal of the Association for Research in Otolaryngology	2016
Expert Opinions on Managing Fertility and Pregnancy in Patients With Mucopolysaccharidosis F Stewart, A Bentley, BK Burton, N Guffon, SL Hale, PR Harmatz, SG Kircher, PK Kochhar, JJ Mitchell, U Plöckinger, J Semotok, S Graham, S Sande, Z Sisic, TA Johnston	Journal of Inborn Errors of Metabolism and Screening	2016
The effect of Tlr4 and/or C3 deficiency and of neonatal gene therapy on skeletal disease in mucopolysaccharidosis VII mice EM Xing, S Wu, KP Ponder	Molecular Genetics and Metabolism	2015
Mucopolysaccharidosis VII in a Cat Caused by 2 Adjacent Missense Mutations in the GUSB Gene P Wang, J Sorenson, S Strickland, C Mingus, ME Haskins, U Giger	Journal of Veterinary Internal Medicine	2015
Rosenberg's Molecular and Genetic Basis of Neurological and Psychiatric Disease R Schiffmann	Rosenberg's Molecular and Genetic Basis of Neurological and Psychiatric Disease	2015
Structure and function of the interphotoreceptor matrix surrounding retinal photoreceptor cells M Ishikawa, Y Sawada, T Yoshitomi	Experimental Eye Research	2015
“Partial rescue of Mucopolysaccharidosis Type VII mice with a lifelong engraftment of allogeneic stem cells in utero”: IUHCT improves phenotypes of MPSII mice N Ihara, A Umezawa, N Onami, H Tsumura, E Inoue, S Hayashi, H Sago, S Mizutani		2014
Central Nervous System Delivery of Helper-Dependent Canine Adenovirus Corrects Neuropathology and Behavior in Mucopolysaccharidosis Type VII Mice L Ariza, L Giménez-Llort, A Cubizolle, G Pagès, B García-Lareu, N Serratrice, D Cots, R Thwaite, M Chillón, EJ Kremer, A Bosch	Human Gene Therapy	2014
Lentiviral-Mediated Gene Therapy Results in Sustained Expression of β-Glucuronidase for up to 12 Months in the Gus mps/mps and up to 18 Months in the Gus tm(L175F)Sly Mouse Models of Mucopolysaccharidosis Type VII AL Derrick-Roberts, CE Pyragius, XM Kaidonis, MR Jackson, DS Anson, S Byers	Human Gene Therapy	2014
Autophagy in Health and Disease M Gurney, G Muralidhar, PJ Linton	Autophagy in Health and Disease	2013
Gene Therapy S Nimesh	Gene Therapy	2013
Magnetic resonance findings in a domestic short‐hair cat with presumptive mucopolysaccharidosis R Fernandes, GB Cherubini, A Caine, V Palus	Veterinary Record Case Reports	2013
Long circulating enzyme replacement therapy rescues bone pathology in mucopolysaccharidosis VII murine model DJ Rowan, S Tomatsu, JH Grubb, B Haupt, AM Montaño, H Oikawa, AC Sosa, A Chen, WS Sly	Molecular Genetics and Metabolism	2012
Skeletal response to lentiviral mediated gene therapy in a mouse model of MPS VII CE Macsai, AL Derrick-Roberts, X Ding, KH Zarrinkalam, C McIntyre, PH Anderson, DS Anson, S Byers	Molecular Genetics and Metabolism	2012
Assessment of bone dysplasia by micro-CT and glycosaminoglycan levels in mouse models for mucopolysaccharidosis type I, IIIA, IVA, and VII DJ Rowan, S Tomatsu, JH Grubb, AM Montaño, WS Sly	Journal of Inherited Metabolic Disease	2012
Glycosaminoglycan Storage Disorders: A Review MF Coutinho, L Lacerda, S Alves	Biochemistry Research International	2012
A Novel GUSB Mutation in Brazilian Terriers with Severe Skeletal Abnormalities Defines the Disease as Mucopolysaccharidosis VII MK Hytönen, M Arumilli, AK Lappalainen, H Kallio, M Snellman, K Sainio, H Lohi, B Dermaut	PloS one	2012
Dysregulation of Gene Expression in a Lysosomal Storage Disease Varies between Brain Regions Implicating Unexpected Mechanisms of Neuropathology MK Parente, R Rozen, CN Cearley, JH Wolfe, R Klein	PloS one	2012
Biotransformation of dehydroepiandrosterone withMacrophomina phaseolinaand β-glucuronidase inhibitory activity of transformed products M. Iqbal Choudhary, Salman Zafar, Naik Tameen Khan, Saeed Ahmad, Shagufta Noreen, Bishnu P. Marasini, Abdulaziz A. Al-Khedhairy, Atta-ur-Rahman	Journal of Enzyme Inhibition and Medicinal Chemistry	2011
From Serendipity to Therapy EF Neufeld	Annual Review of Biochemistry	2011
Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation D Wang, C Shukla, X Liu, TR Schoeb, LA Clarke, DM Bedwell, KM Keeling	Molecular Genetics and Metabolism	2010
Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy SC Jung, ES Park, EN Choi, CH Kim, SJ Kim, DK Jin	Molecules and Cells	2010
Why are behaviors of children suffering from various neuronopathic types of mucopolysaccharidoses different? G Węgrzyn, J Jakóbkiewicz-Banecka, M Narajczyk, A Wiśniewski, E Piotrowska, M Gabig-Cimińska, A Kloska, M Słomińska-Wojewódzka, A Korzon-Burakowska, A Węgrzyn	Medical Hypotheses	2010
Pulmonary surfactant: an immunological perspective ZC Chroneos, Z Sever-Chroneos, VL Shepherd	Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology	2009
Mechanism of shortened bones in mucopolysaccharidosis VII JA Metcalf, Y Zhang, MJ Hilton, F Long, KP Ponder	Molecular Genetics and Metabolism	2009
Comparative pathology of murine mucolipidosis types II and IIIC P Vogel, BJ Payne, R Read, WS Lee, CM Gelfman, S Kornfeld	Veterinary pathology	2009
Altered lumbar spine structure, biochemistry, and biomechanical properties in a canine model of mucopolysaccharidosis type VII LJ Smith, JT Martin, SE Szczesny, KP Ponder, ME Haskins, DM Elliott	Journal of orthopaedic research : official publication of the Orthopaedic Research Society	2009
Glycosaminoglycan-mediated loss of cathepsin K collagenolytic activity in MPS I contributes to osteoclast and growth plate abnormalities S Wilson, S Hashamiyan, L Clarke, P Saftig, J Mort, VM Dejica, D Brömme	The American Journal of Pathology	2009
Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome) S Tomatsu, AM Montaño, VC Dung, JH Grubb, WS Sly	Human Mutation	2009
Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery M Richard, A Arfi, J Seguin, C Gandolphe, D Scherman	Gene Therapy	2009
Gene Therapy for Lysosomal Storage Diseases (LSDs) in Large Animal Models M Haskins	ILAR journal / National Research Council, Institute of Laboratory Animal Resources	2009
Esterase 22 and beta-glucuronidase hydrolyze retinoids in mouse liver R Schreiber, U Taschler, H Wolinski, A Seper, SN Tamegger, M Graf, SD Kohlwein, G Haemmerle, R Zimmermann, R Zechner, A Lass	Journal of lipid research	2009
Widespread nonhematopoietic tissue distribution by transplanted human progenitor cells with high aldehyde dehydrogenase activity DA Hess, TP Craft, L Wirthlin, S Hohm, P Zhou, WC Eades, MH Creer, MS Sands, JA Nolta	Stem Cells	2008
CNS-directed gene therapy for lysosomal storage diseases MS Sands, ME Haskins	Acta Paediatrica (Oslo, Norway : 1992.) Supplement	2008
Transplantation and magnetic resonance imaging of canine neural progenitor cell grafts in the postnatal dog brain RM Walton, SG Magnitsky, GS Seiler, H Poptani, JH Wolfe	Journal of Neuropathology and Experimental Neurology	2008
Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy RS Herati, VW Knox, P O'Donnell, M D'Angelo, ME Haskins, KP Ponder	Molecular Genetics and Metabolism	2008
Lentiviral-transduced human mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplantation model of human disease TE Meyerrose, M Roberts, KK Ohlemiller, CA Vogler, L Wirthlin, JA Nolta, MS Sands	Stem Cells	2008
Identification of new markers for neurodegeneration process in the mouse model of Sly disease as revealed by expression profiling of selected genes M Richard, A Arfı, H Rhinn, C Gandolphe, D Scherman	Journal of Neuroscience Research	2008
Clinical Biochemistry of Domestic Animals BC Tennant, SA Center	Clinical Biochemistry of Domestic Animals	2008
Artificial cells, cell engineering and therapy M Potter, A Li, P Cirone, F Shen, P Chang	Artificial cells, cell engineering and therapy	2007
Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses EL Aronovich, JB Bell, LR Belur, R Gunther, B Koniar, DC Erickson, PA Schachern, I Matise, RS McIvor, CB Whitley, PB Hackett	The Journal of Gene Medicine	2007
The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome) AR Garcia, J Pan, JC Lamsa, J Muenzer	Journal of Inherited Metabolic Disease	2007
Clinical response to persistent, low-level β-glucuronidase expression in the murine model of mucopolysaccharidosis type VII A Donsante, B Levy, C Vogler, MS Sands	Journal of Inherited Metabolic Disease	2007
Lysosomal Enzyme Replacement of the Brain with Intravenous Non-Viral Gene Transfer Y Zhang, Y Wang, RJ Boado, WM Pardridge	Pharmaceutical Research	2007
Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II AR Garcia, JM DaCosta, J Pan, J Muenzer, JC Lamsa	Molecular Genetics and Metabolism	2007
Animal models for mucopolysaccharidosis disorders and their clinical relevance ME Haskins	Acta Paediatrica (Oslo, Norway : 1992.) Supplement	2007
Tissue refractometry using Hilbert phase microscopy N Lue, J Bewersdorf, MD Lessard, K Badizadegan, RR Dasari, MS Feld, G Popescu	Optics Letters	2007
A Single Injection of an Adeno-Associated Virus Vector into Nuclei with Divergent Connections Results in Widespread Vector Distribution in the Brain and Global Correction of a Neurogenetic Disease CN Cearley, JH Wolfe	The Journal of neuroscience : the official journal of the Society for Neuroscience	2007
Lysosomal Storage Disorders JA Barranger, MA Cabrera-Salazar		2007
Gene targeting in vivo by adeno-associated virus vectors DG Miller, PR Wang, LM Petek, RK Hirata, MS Sands, DW Russell	Nature Biotechnology	2006
Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs B Wang, TM O’Malley, L Xu, C Vite, P Wang, PA O’Donnell, NM Ellinwood, ME Haskins, KP Ponder	Molecular Genetics and Metabolism	2006
Attenuation of murine lysosomal storage disease by allogeneic neonatal bone marrow transplantation using costimulatory blockade and donor lymphocyte infusion without myeloablation MD Lessard, TL Alley, JL Proctor, B Levy, N Galvin, CA Vogler, BW Soper	Clinical Immunology	2006
Characterization of a C57BL/6 congenic mouse strain of mucopolysaccharidosis type IIIA AC Crawley, BL Gliddon, D Auclair, SL Brodie, C Hirte, BM King, M Fuller, KM Hemsley, JJ Hopwood	Brain Research	2006
Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain CN Cearley, JH Wolfe	Molecular Therapy	2006
Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII C Vogler, B Levy, JH Grubb, N Galvin, Y Tan, E Kakkis, N Pavloff, WS Sly	Proceedings of the National Academy of Sciences	2005
Large animal models and gene therapy M Casal, M Haskins	European Journal of Human Genetics	2005
Early Onset of Lysosomal Storage Disease in a Murine Model of Mucopolysaccharidosis Type VII: Undegraded Substrate Accumulates in Many Tissues in the Fetus and Very Young MPS VII Mouse C Vogler, B Levy, N Galvin, M Lessard, B Soper, J Barker	Pediatric and Developmental Pathology	2005
Α genetic fusion construct between the tetanus toxin C fragment and the lysosomal acid hydrolase β-glucuronidase expresses a bifunctional protein with enhanced secretion and neuronal uptake K Jiang, DJ Watson, JH Wolfe	Journal of Neurochemistry	2005
International Review of Neurobiology ZT Bloomgarden	International review of neurobiology	2005
Laboratory Techniques in Biochemistry and Molecular Biology S Chen, KM Madsen, CC Tisher, A Agarwal	Laboratory Techniques in Biochemistry and Molecular Biology	2005
Neurological Disease and Therapy: Scientific and Clinical Advances S Perlman	Neurological Disease and Therapy: Principles and Controversies	2005
Development of MPS IVA mouse (Galnstm(hC79S·mC76S)slu) tolerant to human N-acetylgalactosamine-6-sulfate sulfatase S Tomatsu, M Gutierrez, T Nishioka, M Yamada, M Yamada, Y Tosaka, JH Grubb, AM Montaño, MB Vieira, GG Trandafirescu, OM Peña, S Yamaguchi, KO Orii, T Orii, A Noguchi, L Laybauer	Human Molecular Genetics	2005
Stable Levels of Long-Term Transgene Expression Driven by the Latency-Associated Transcript Promoter in a Herpes Simplex Virus Type 1 Vector BK Berges, JH Wolfe, NW Fraser	Molecular Therapy	2005
Functional Correction of CNS Phenotypes in a Lysosomal Storage Disease Model Using Adeno-Associated Virus Type 4 Vectors G Liu	The Journal of neuroscience : the official journal of the Society for Neuroscience	2005
Neurodegenerative Diseases: Neurobiology, Pathogenesis and Therapeutics MF Beal, AE Lang, AC Ludolph		2005
Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII JC Woloszynek, M Roberts, T Coleman, C Vogler, W Sly, CF Semenkovich, MS Sands	Biochemical Journal	2004
Widespread Correction of Lysosomal Storage Following Intrahepatic Injection of a Recombinant Adeno-associated Virus in the Adult MPS VII Mouse TJ Sferra, K Backstrom, C Wang, R Rennard, M Miller, Y Hu	Molecular Therapy	2004
Widespread gene transduction to the central nervous system by adenovirusin utero: implication for prenatal gene therapy to brain involvement of lysosomal storage disease JS Shen, XL Meng, H Maeda, T Ohashi, Y Eto	The Journal of Gene Medicine	2004
Excision of Sleeping Beauty transposons: parameters and applications to gene therapy G Liu, EL Aronovich, Z Cui, CB Whitley, PB Hackett	The Journal of Gene Medicine	2004
Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice JH LeBowitz, JH Grubb, JA Maga, DH Schmiel, C Vogler, WS Sly	Proceedings of the National Academy of Sciences	2004
Electrocardiographic and other cardiac anomalies in beta-glucuronidase-null mice corrected by nonablative neonatal marrow transplantation AJ Schuldt, TJ Hampton, V Chu, CA Vogler, N Galvin, MD Lessard, JE Barker	Proceedings of the National Academy of Sciences	2004
Mucopolysaccharidosis type VII in a German Shepherd Dog DC Dombrowski, KP Carmichael, P Wang, TM O'Malley, ME Haskins, U Giger	Journal of the American Veterinary Medical Association	2004
Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders R McGlynn, K Dobrenis, SU Walkley	The Journal of Comparative Neurology	2004
Prevention of neuropathology in the mouse model of Hurler syndrome N Desmaris, L Verot, JP Puech, C Caillaud, MT Vanier, JM Heard	Annals of Neurology	2004
Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease AA Hofling, S Devine, C Vogler, MS Sands	Molecular Therapy	2004
Brain transplantation of genetically modified bone marrow stromal cells corrects CNS pathology and cognitive function in MPS VII mice K Sakurai, S Iizuka, JS Shen, XL Meng, T Mori, A Umezawa, T Ohashi, Y Eto	Gene Therapy	2004
In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice L Xu, T O'Malley, MS Sands, B Wang, T Meyerrose, ME Haskins, KP Ponder	Molecular Therapy	2004
AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice AK Hennig, JM Ogilvie, KK Ohlemiller, AM Timmers, WW Hauswirth, MS Sands	Molecular Therapy	2004
Treatment of lysosomal storage disorders: Cell therapy and gene therapy Y Eto, , , T Ohashi	Journal of Inherited Metabolic Disease	2004
Gene therapy for lysosomal storage disorders YA Ioannou, A Enriquez, C Benjamin	Expert Opinion on Biological Therapy	2003
Production of MPS VII mouse (Gus(tm(hE540A x mE536A)Sly)) doubly tolerant to human and mouse beta-glucuronidase S Tomatsu, KO Orii, C Vogler, JH Grubb, EM Snella, M Gutierrez, T Dieter, CC Holden, K Sukegawa, T Orii, N Kondo, WS Sly	Human Molecular Genetics	2003
Biodistribution and efficacy of donor T lymphocytes in a murine model of lysosomal storage disease PP Young, C Vogler, AA Hofling, MS Sands	Molecular Therapy	2003
Improvement of skeletal lesions in mice with mucopolysaccharidosis type vii by neonatal adenoviral gene transfer A Kanaji, M Kosuga, XK Li, Y Fukuhara, A Tanabe, Y Kamata, N Azuma, M Yamada, T Sakamaki, Y Toyama, T Okuyama	Molecular Therapy	2003
Brain transplantation of genetically engineered human neural stem cells globally corrects brain lesions in the mucopolysaccharidosis type VII mouse XL Meng, JS Shen, T Ohashi, H Maeda, SU Kim, Y Eto	Journal of Neuroscience Research	2003
Long-term normalization in the central nervous system, ocular manifestations and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII Y Kamata, A Tanabe, A Kanaji, M Kosuga, Y Fukuhara, XK Li, S Suzuki, M Yamada, N Azuma, T Okuyama	Gene Therapy	2003
Neonatal or hepatocyte growth factor–potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B L Xu, C Gao, MS Sands, SR Cai, TC Nichols, DA Bellinger, RA Raymer, S McCorquodale, KP Ponder	Blood	2003
Engraftment of human CD34+ cells leads to widespread distribution of donor-derived cells and correction of tissue pathology in a novel murine xenotransplantation model of lysosomal storage disease AA Hofling, C Vogler, MH Creer, MS Sands	Blood	2003
Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice Anne K Hennig, Beth Levy, Judith Mosinger Ogilvie, Carole A Vogler, Nancy Galvin, Steven Bassnett, Mark S Sands	The Journal of neuroscience : the official journal of the Society for Neuroscience	2003
Intraventricular Brain Injection of Adeno-Associated Virus Type 1 (AAV1) in Neonatal Mice Results in Complementary Patterns of Neuronal Transduction to AAV2 and Total Long-Term Correction of Storage Lesions in the Brains of β-Glucuronidase-Deficient Mice MA Passini, DJ Watson, CH Vite, DJ Landsburg, AL Feigenbaum, JH Wolfe	Journal of virology	2003
Successful Allogeneic Neonatal Bone Marrow Transplantation Devoid of Myeloablation Requires Costimulatory Blockade BW Soper, MD Lessard, CD Jude, AJ Schuldt, RM Bunte, JE Barker	Journal of immunology (Baltimore, Md. : 1950)	2003
Intravitreal Gene Therapy Reduces Lysosomal Storage in Specific Areas of the CNS in Mucopolysaccharidosis VII Mice Hennig AK, Levy B, Ogilvie JM, Vogler CA, Galvin N, Bassnett S, Sands MS	Journal of Neuroscience	2003
Animal models for mucopolysaccharidoses and their clinical relevance M Haskins, M Casal, NM Ellinwood, J Melniczek, H Mazrier, U Giger	Acta Paediatrica	2002
THEFELINEGENOMEPROJECT1 SJ O'Brien, M Menotti-Raymond, WJ Murphy, N Yuhki	Annual Review of Genetics	2002
Enzyme replacement therapy: from concept to clinical practice WS Sly	Acta Paediatrica	2002
Missense models [Gustm(E536A)Sly, Gustm(E536Q)Sly, and Gustm(L175F)Sly] of murine mucopolysaccharidosis type VII produced by targeted mutagenesis S Tomatsu, KO Orii, C Vogler, JH Grubb, EM Snella, MA Gutierrez, T Dieter, K Sukegawa, T Orii, N Kondo, WS Sly	Proceedings of the National Academy of Sciences	2002
Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier WS Sly, C Vogler	Proceedings of the National Academy of Sciences	2002
Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors AI Brooks, CS Stein, SM Hughes, J Heth, PM McCray, SL Sauter, JC Johnston, DA Cory-Slechta, HJ Federoff, BL Davidson	Proceedings of the National Academy of Sciences	2002
Feline immunodeficiency virus vectors. Gene transfer to mouse retina following intravitreal injection TA Derksen, SL Sauter, BL Davidson	The Journal of Gene Medicine	2002
Delayed administration of carrier marrow can decrease competition on donor stem cells during engraftment and maintain radioprotection of the host BW Soper, MD Lessard, CD Jude, AJ Schuldt, JE Barker	Experimental Hematology	2002
Inner ear pathology in the mucopolysaccharidosis VII mouse KK Ohlemiller, AK Hennig, JM Lett, AF Heidbreder, MS Sands	Hearing Research	2002
Long-Term Expression of β-Glucuronidase by Genetically Modified Human Neural Progenitor Cells Grafted into the Mouse Central Nervous System D Buchet, C Serguera, V Zennou, P Charneau, J Mallet	Molecular and Cellular Neuroscience	2002
Abnormal osteoclast morphology and bone remodeling in a murine model of a lysosomal storage disease M A Monroy, F P Ross, S L Teitelbaum, M S Sands	Bone	2002
Evaluation of Pathological Manifestations of Disease in Mucopolysaccharidosis VII Mice after Neonatal Hepatic Gene Therapy L Xu, RL Mango, MS Sands, ME Haskins, NM Ellinwood, KP Ponder	Molecular Therapy	2002
Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible GG Heuer, MA Passini, K Jiang, MK Parente, VM Lee, JQ Trojanowski, JH Wolfe	Annals of Neurology	2002
Enhanced Secretion and Uptake of β-Glucuronidase Improves Adeno-associated Viral-Mediated Gene Therapy of Mucopolysaccharidosis Type VII Mice SS Elliger, CA Elliger, C Lang, GL Watson	Molecular Therapy	2002
Modification of multiple transcriptional regulatory elements in a Moloney murine leukemia virus gene transfer vector circumvents silencing in fibroblast grafts and increases levels of expression of the transferred enzyme RK Alur, B Foley, MK Parente, DK Tobin, GG Heuer, AN Avadhani, J Pongubala, JH Wolfe	Gene Therapy	2002
Lentiviral Vectors D Trono		2002
Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream Marco A Passini, Edward B Lee, Gregory G Heuer, John H Wolfe	The Journal of neuroscience : the official journal of the Society for Neuroscience	2002
Distribution of a Lysosomal Enzyme in the Adult Brain by Axonal Transport and by Cells of the Rostral Migratory Stream Passini MA, Lee EB, Heuer GG, Wolfe JH	Journal of Neuroscience	2002
Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice WS Sly, C Vogler, JH Grubb, M Zhou, J Jiang, XY Zhou, S Tomatsu, Y Bi, EM Snella	Proceedings of the National Academy of Sciences	2001
In Utero Fetal Liver Cell Transplantation without Toxic Irradiation Alleviates Lysosomal Storage in Mice with Mucopolysaccharidosis Type VII JE Barker, S Deveau, M Lessard, N Hamblen, C Vogler, B Levy	Blood Cells, Molecules, and Diseases	2001
From gene transfer to gene therapy in lysosomal storage diseases affecting the central nervous system L Poenaru	Annals of Medicine	2001
A Novel Model of Murine Mucopolysaccharidosis Type VII due to an Intracisternal A Particle Element Transposition into the β-Glucuronidase Gene: Clinical and Pathologic Findings C Vogler, B Levy, N Galvin, MS Sands, EH Birkenmeier, WS Sly, J Barker	Pediatric Research	2001
Nonablative neonatal marrow transplantation attenuates functional and physical defects of beta-glucuronidase deficiency BW Soper, MD Lessard, CA Vogler, B Levy, WG Beamer, WS Sly, JE Barker	Blood	2001
Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer TM Daly, KK Ohlemiller, MS Roberts, CA Vogler, MS Sands	Gene Therapy	2001
Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII WA Frisella, LH O'Connor, CA Vogler, M Roberts, S Walkley, B Levy, TM Daly, MS Sands	Molecular Therapy	2001
Accumulation of Abnormal Amounts of Glycosaminoglycans in Murine Mucopolysaccharidosis Type VII Neural Progenitor Cells Does Not Alter the Growth Rate or Efficiency of Differentiation into Neurons GG Heuer, AF Skorupa, RK Alur, K Jiang, JH Wolfe	Molecular and Cellular Neuroscience	2001
Adenovirus-Mediated Gene Therapy for Corneal Clouding in Mice with Mucopolysaccharidosis Type VII Y Kamata, T Okuyama, M Kosuga, A O'hira, A Kanaji, K Sasaki, M Yamada, N Azuma	Molecular Therapy	2001
Primary Adult Human Astrocytes as an ex Vivo Vehicle for β-Glucuronidase Delivery in the Brain C Serguera, C Sarkis, JL Ridet, P Colin, P Moullier, J Mallet	Molecular Therapy	2001
Biodistribution, Kinetics, and Efficacy of Highly Phosphorylated and Non-phosphorylated β-Glucuronidase in the Murine Model of Mucopolysaccharidosis VII MS Sands, CA Vogler, KK Ohlemiller, MS Roberts, JH Grubb, B Levy, WS Sly	The Journal of biological chemistry	2001
Engraftment of Genetically Engineered Amniotic Epithelial Cells Corrects Lysosomal Storage in Multiple Areas of the Brain in Mucopolysaccharidosis Type VII Mice M Kosuga, K Sasaki, A Tanabe, XK Li, H Okawa, I Ogino, O Okuda, H Arai, N Sakuragawa, Y Kamata, N Azuma, S Suzuki, M Yamada, T Okuyama	Molecular Therapy	2001
Production and Neurotropism of Lentivirus Vectors Pseudotyped with Lyssavirus Envelope Glycoproteins N Desmaris, A Bosch, C Salaün, C Petit, MC Prévost, N Tordo, P Perrin, O Schwartz, H Rocquigny, JM Heard	Molecular Therapy	2001
Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors A Donsante, C Vogler, N Muzyczka, JM Crawford, J Barker, T Flotte, M Campbell-Thompson, T Daly, MS Sands	Gene Therapy	2001
In utero transplantation of fetal liver cells in the mucopolysaccharidosis type VII mouse results in low-level chimerism, but overexpression of β-glucuronidase can delay onset of clinical signs ML Casal, JH Wolfe	Blood	2001
Treatment of a Lysosomal Storage Disease, Mucopolysaccharidosis VII, with Microencapsulated Recombinant Cells CJ Ross, L Bastedo, SA Maier, MS Sands, PL Chang	Human Gene Therapy	2000
Recombinant Adeno-Associated Virus-Mediated Correction of Lysosomal Storage within the Central Nervous System of the Adult Mucopolysaccharidosis Type VII Mouse TJ Sferra, G Qu, D McNeely, R Rennard, KR Clark, WD Lo, PR Johnson	Human Gene Therapy	2000
Novel Upf2p orthologues suggest a functional link between translation initiation and nonsense surveillance complexes JT Mendell, SM Medghalchi, RG Lake, EN Noensie, HC Dietz	Molecular and cellular biology	2000
Retinal Function is Improved in a Murine Model of a Lysosomal Storage Disease Following Bone Marrow Transplantation KK Ohlemiller, CA Vogler, M Roberts, N Galvin, MS Sands	Experimental Eye Research	2000
Short-Term Enzyme Replacement in the Murine Model of Sanfilippo Syndrome Type B WH Yu, KW Zhao, S Ryazantsev, N Rozengurt, EF Neufeld	Molecular Genetics and Metabolism	2000
Reversal of Pathology in the Entire Brain of Mucopolysaccharidosis Type VII Mice after Lentivirus-Mediated Gene Transfer A Bosch, E Perret, N Desmaris, D Trono, JM Heard	Human Gene Therapy	2000
Abnormal Immune Function In Vivo in a Murine Model of Lysosomal Storage Disease TM Daly, RG Lorenz, MS Sands	Pediatric Research	2000
Mucopolysaccharidosis Type VII in the Developing Mouse Fetus ML Casal, JH Wolfe	Pediatric Research	2000
Somatic gene therapy for a neurodegenerative disease using microencapsulated recombinant cells CJ Ross, M Ralph, PL Chang	Experimental Neurology	2000
Systemic hyperosmolality improves beta-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer A Ghodsi, C Stein, T Derksen, I Martins, RD Anderson, BL Davidson	Experimental Neurology	2000
Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII JH Wolfe, MS Sands, N Harel, MA Weil, MK Parente, AC Polesky, JJ Reilly, C Hasson, S Weimelt, ME Haskins	Molecular Therapy	2000
The mouse N-acetylgalactosamine-6-sulfate sulfatase (Galns) gene: cDNA isolation, genomic characterization, chromosomal assignment and analysis of the 5'-flanking region A M Montaño, A Yamagishi, S Tomatsu, S Fukuda, N G Copeland, K E Orii, K Isogai, N Yamada, Z I Kato, N A Jenkins, D J Gilbert, K Sukegawa, T Orii, N Kondo	Biochimica et Biophysica Acta	2000
Long-Term and Significant Correction of Brain Lesions in Adult Mucopolysaccharidosis Type VII Mice Using Recombinant AAV Vectors A Bosch, E Perret, N Desmaris, JM Heard	Molecular Therapy	2000
Delivery of a Retroviral Vector Expressing Human β-Glucuronidase to the Liver and Spleen Decreases Lysosomal Storage in Mucopolysaccharidosis VII Mice C Gao, MS Sands, ME Haskins, KP Ponder	Molecular Therapy	2000
Adenovirus-Mediated Gene Therapy for Mucopolysaccharidosis VII: Involvement of Cross-Correction in Wide-Spread Distribution of the Gene Products and Long-Term Effects of CTLA-4Ig Coexpression M Kosuga, S Takahashi, K Sasaki, XK Li, M Fujino, H Hamada, S Suzuki, M Yamada, N Matsuo, T Okuyama	Molecular Therapy	2000
Gene therapy in the CNS LC Costantini, JC Bakowska, XO Breakefield, O Isacson	Gene Therapy	2000
Significantly Increased Expression of β-Glucuronidase in the Central Nervous System of Mucopolysaccharidosis Type VII Mice from the Latency-Associated Transcript Promoter in a Nonpathogenic Herpes Simplex Virus Type 1 Vector J Zhu, W Kang, JH Wolfe, NW Fraser	Molecular Therapy	2000
Defects in the cappuccino (cno) gene on mouse chromosome 5 and human 4p cause Hermansky-Pudlak syndrome by an AP-3–independent mechanism B Gwynn, SL Ciciotte, SJ Hunter, LL Washburn, RS Smith, SG Andersen, RT Swank, EC Dell'Angelica, JS Bonifacino, EM Eicher, LL Peters	Blood	2000
Reduction of lysosomal storage in murine mucopolysaccharidosis type VII by transplantation of normal and genetically modified macrophages T Ohashi, T Yokoo, S Iizuka, H Kobayashi, WS Sly, Y Eto	Blood	2000
Neonatal Intramuscular Injection with Recombinant Adeno-Associated Virus Results in Prolonged beta-Glucuronidase Expression in Situ and Correction of Liver Pathology in Mucopolysaccharidosis Type VII Mice TM Daly, T Okuyama, C Vogler, ME Haskins, N Muzyczka, MS Sands	Human Gene Therapy	1999
Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice CS Stein, A Ghodsi, T Derksen, BL Davidson	Journal of virology	1999
Encapsulation for Somatic Gene Therapy PL Chang	Annals of the New York Academy of Sciences	1999
Growth hormone and insulin-like growth factor-I enhance β-glucuronidase gene activation by androgen in mouse kidney GL Niermann, GL Watson	Molecular and Cellular Endocrinology	1999
Sustained Production of β-Glucuronidase from Localized Sites after AAV Vector Gene Transfer Results in Widespread Distribution of Enzyme and Reversal of Lysosomal Storage Lesions in a Large Volume of Brain in Mucopolysaccharidosis VII Mice AF Skorupa, KJ Fisher, JM Wilson, MK Parente, JH Wolfe	Experimental Neurology	1999
Enzyme Replacement Therapy Improves Reproductive Performance in Mucopolysaccharidosis Type VII Mice But Does Not Prevent Postnatal Losses BW Soper, AW Pung, CA Vogler, JH Grubb, WS Sly, JE Barker	Pediatric Research	1999
Enzyme Replacement in Murine Mucopolysaccharidosis Type VII: Neuronal and Glial Response to β-Glucuronidase Requires Early Initiation of Enzyme Replacement Therapy C Vogler, B Levy, NJ Galvin, C Thorpe, MS Sands, JE Barker, J Baty, EH Birkenmeier, WS Sly	Pediatric Research	1999
Molecular Basis of Feline β-Glucuronidase Deficiency: An Animal Model of Mucopolysaccharidosis VII JC Fyfe, RL Kurzhals, ME Lassaline, PS Henthorn, PR Alur, P Wang, JH Wolfe, U Giger, ME Haskins, DF Patterson, H Sun, S Jain, N Yuhki	Genomics	1999
Causal Association between Receptivity to Tobacco Promotions and Smoking in Adolescents JD Sargent, MA Dalton, ML Beach, AM Bernhardt, M Stevens	Pediatric Research	1999
Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector SS Elliger, CA Elliger, CP Aguilar, NR Raju, GL Watson	Gene Therapy	1999
Enhanced In Situ Detection of β-Glucuronidase Activity in Murine Tissue GC Kopen, DJ Prockop, DG Phinney	Journal of Histochemistry & Cytochemistry	1999
Single Cell Analysis and Selection of Living Retrovirus Vector-corrected Mucopolysaccharidosis VII Cells Using a Fluorescence-activated Cell Sorting-based Assay for Mammalian β-Glucuronidase Enzymatic Activity MC Lorincz, MK Parente, M Roederer, GP Nolan, Z Diwu, DI Martin, LA Herzenberg, JH Wolfe	The Journal of biological chemistry	1999
Mouse Models in the Study of Genetic Neurological Disorders B Popko		1999
Cell Encapsulation Technology and Therapeutics WM Kühtreiber, RP Lanza, WL Chick		1999
Behavior and Therapeutic Efficacy of β-Glucuronidase–Positive Mononuclear Phagocytes in a Murine Model of Mucopolysaccharidosis Type VII BJ Freeman, MS Roberts, CA Vogler, A Nicholes, AA Hofling, MS Sands	Blood	1999
Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function LH O'Connor, LC Erway, CA Vogler, WS Sly, A Nicholes, J Grubb, SW Holmberg, B Levy, MS Sands	Journal of Clinical Investigation	1998
Extensiveβ-Glucuronidase Activity in Murine Central Nervous System after Adenovirus-Mediated Gene Transfer to Brain A Ghodsi, C Stein, T Derksen, G Yang, RD Anderson, BL Davidson	Human Gene Therapy	1998
Sulfamidase Deficiency in a Family of Dachshunds: A Canine Model of Mucopolysaccharidosis IIIA (Sanfilippo A) A Fischer, KP Carmichael, JF Munnell, P Jhabvala, JN Thompson, R Matalon, PF Jezyk, P Wang, U Giger	Pediatric Research	1998
Gene therapy for lysosomal storage diseases TM Daly, MS Sands	Expert Opinion on Investigational Drugs	1998
Efficient and persistent expression of β-glucuronidase gene in CD34+ cells from human umbilical cord blood by retroviral vector T Ohashi, S Iizuka, WS Sly, K Machiki, Y Eto	European Journal of Haematology	1998
Murine MPS I: insights into the pathogenesis of Hurler syndrome C Russell, G Hendson, G Jevon, T Matlock, J Yu, M Aklujkar, KY Ng, LA Clarke	Clinical Genetics	1998
Mouse Models of Human Lysosomal Diseases K Suzuki, RL Proia, K Suzuki	Brain Pathology	1998
Cloning of the Canine β-Glucuronidase cDNA, Mutation Identification in Canine MPS VII, and Retroviral Vector-Mediated Correction of MPS VII Cells J Ray, A Bouvet, C DeSanto, JC Fyfe, D Xu, JH Wolfe, GD Aguirre, DF Patterson, ME Haskins, PS Henthorn	Genomics	1998
Murine mucopolysaccharidosis type VII: The impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease C Vogler, MS Sands, N Galvin, B Levy, C Thorpe, J Barker, WS Sly	Journal of Inherited Metabolic Disease	1998
Intracisternal A-Particle Element Transposition into the Murine β-Glucuronidase Gene Correlates with Loss of Enzyme Activity: a New Model for β-Glucuronidase Deficiency in the C3H Mouse B Gwynn, K Lueders, MS Sands, EH Birkenmeier	Molecular and cellular biology	1998
Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation MS Sands, C Vogler, A Torrey, B Levy, B Gwynn, J Grubb, WS Sly, EH Birkenmeier	Journal of Clinical Investigation	1997
Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice T Ohashi, K Watabe, K Uehara, WS Sly, C Vogler, Y Eto	Proceedings of the National Academy of Sciences	1997
Gene therapy for lysosomal storage disease: A no-brainer? WS Sly, C Vogler	Nature Medicine	1997
Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of β-glucuronidase R Taylor, J Wolfe	Nature Medicine	1997
Neural Precursor Cells: Applications for the Study and Repair of the Central Nervous System LJ Fisher	Neurobiology of Disease	1997
Microcapsules as Bio-organs for Somatic Gene Therapya PL Chang	Annals of the New York Academy of Sciences	1997
The Use of Nonneuronal Cells for Gene Delivery EY Snyder, MC Senut	Neurobiology of Disease	1997
Adenovirus-mediated gene transfer and expression of human -glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice T Ohashi, K Watabe, K Uehara, WS Sly, C Vogler, Y Eto	Proceedings of the National Academy of Sciences	1997
Lysosomal Storage Diseases of Animals: An Essay in Comparative Pathology RD Jolly, SU Walkley	Veterinary pathology	1997
The interphotoreceptor matrix, a space in sight K Mieziewska	Microscopy Research and Technique	1996
Long-Term Secretion of Therapeutic Proteins from Genetically Modified Skeletal Muscles N Naffakh, C Pinset, D Montarras, Z Li, D Paulin, O Danos, JM Heard	Human Gene Therapy	1996
Enzyme Replacement with Recombinant β-Glucuronidase in Murine Mucopolysaccharidosis Type VII: Impact of Therapy during the First Six Weeks of Life on Subsequent Lysosomal Storage, Growth, and Survival C Vogler, MS Sands, B Levy, N Galvin, EH Birkenmeier, WS Sly	Pediatric Research	1996
Microencapsulation — An alternative approach to gene therapy PL Chang	Transfusion Science	1996
Thérapie génique des maladies lysosomales P Moullier, A Salvetti, D Bohl, O Danos, JM Heard	Archives de Pédiatrie	1996
Molecular Biology of Hematopoiesis 5 NG Abraham, S Asano, G Brittinger, GJ Maestroni, RK Shadduck		1996
Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer T Li, BL Davidson	Proceedings of the National Academy of Sciences	1995
Gene transfer into the intestinal epithelium SJ Henning	Advanced Drug Delivery Reviews	1995
Retroviral Gene Transfer into the Intestinal Epithelium C Lau, HE Soriano, FD Ledley, MJ Finegold, JH Wolfe, EH Birkenmeier, SJ Henning	Human Gene Therapy	1995
Lysosomal storage diseases V Gieselmann	Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease	1995
Neural progenitor cell engraftment corrects lysosomal storage throughout the MRS VII mouse brain EY Snyder, RM Taylor, JH Wolfe	Nature	1995
Growth plate pathology in feline mucopolysaccharidosis VI S Abreu, J Hayden, P Berthold, IM Shapiro, S Decker, D Patterson, M Haskins	Calcified Tissue International	1995
Morphological alterations in dental and periodontal tissues in murine mucopolysaccharidosis type VII A Gritli-Linde, A Linde, M Goldberg	Calcified Tissue International	1995
Enzyme replacement therapy for murine mucopolysaccharidosis type VII MS Sands, C Vogler, JW Kyle, JH Grubb, B Levy, N Galvin, WS Sly, EH Birkenmeier	Journal of Clinical Investigation	1994
Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII L Bastedo, MS Sands, DT Lambert, MA Pisa, E Birkenmeier, PL Chang	Journal of Clinical Investigation	1994
The murine mutation jaundiced is caused by replacement of an arginine with a stop codon in the mRNA encoding the ninth repeat of beta-spectrin ML Bloom, TM Kaysser, CS Birkenmeier, JE Barker	Proceedings of the National Academy of Sciences	1994
Direct plasmid mediated transfection of adult murine brain cells in vivo using cationic liposomes BJ Roessler, BL Davidson	Neuroscience Letters	1994
Feline Mucopolysaccharidosis VII Due to β-glucuronidase Deficiency R Gitzelmann, NU Bosshard, A Superti-Furga, MA Spycher, J Briner, U Wiesmann, H Lutz, B Litschi	Veterinary pathology	1994
Conjugation—Deconjugation Reactions in Drug Metabolism and Toxicity FC Kauffman		1994
Two related proteolipids and dolichol-linked oligosaccharides accumulate in motor neuron degeneration mice (mnd/mnd), a model for neuronal ceroid lipofuscinosis JR Faust, JS Rodman, PF Daniel, JF Dice, RT Bronson	The Journal of biological chemistry	1994
Overexpression rescues the mutant phenotype of L176F mutation causing beta-glucuronidase deficiency mucopolysaccharidosis in two Mennonite siblings BM Wu, S Tomatsu, S Fukuda, K Sukegawa, T Orii, WS Sly	The Journal of biological chemistry	1994
Arylsulfatase B-deficient mucopolysaccharidosis in rats M Yoshida, J Noguchi, H Ikadai, M Takahashi, S Nagase	Journal of Clinical Investigation	1993
Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts P Moullier, D Bohl, JM Heard, O Danos	Nature Genetics	1993
A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII MS Sands, EH Birkenmeier	Proceedings of the National Academy of Sciences	1993
Mutational studies in a patient with the hydrops fetalis form of mucopolysaccharidosis type VII BM Wu, WS Sly	Human Mutation	1993
Gene therapy on the Sly WS Sly	Nature Genetics	1993
Transplantation of adult-derived myoblasts in mice following gene transfer N Naffakh, C Pinset, D Montarras, C Pastoret, O Danos, JM Heard	Neuromuscular Disorders	1993
Pathological characteristics of mucopolysaccharidosis VI in the rat M Yoshida, H Ikadai, A Maekawa, M Takahashi, S Nagaset	Journal of Comparative Pathology	1993
Deconjugation of bilirubin-IX alpha glucuronides: a physiologic role of hepatic microsomal beta-glucuronidase JF Whiting, JP Narciso, V Chapman, BJ Ransil, RT Swank, JL Gollan	The Journal of biological chemistry	1993
A Genetic Linkage Map of the Mouse: Current Applications and Future Prospects NG Copeland, NA Jenkins, DJ Gilbert, JT Eppig, LJ Maltais, JC Miller, WF Dietrich, A Weaver, SE Lincoln, RG Steen, LD Stein, JH Nadeau, ES Lander	Science	1993
N-acetylglucosamine 6-sulphatase deficiency in a Nubian goat: A model of Sanfilippo syndrome type D (mucopolysaccharidosis IIID) JN Thompson, MZ Jones, G Dawson, PS Huffman	Journal of Inherited Metabolic Disease	1992
Herpesvirus vector gene transfer and expression of β–glucuronidase in the central nervous system of MRS VII mice JH Wolfe, SL Deshmane, NW Fraser	Nature Genetics	1992
Gus Protocols JW Kyle, N Galvin, C Vogler, JH Grubb	Gus Protocols	1992
EVOLUTION OF β-GLUCURONIDASE REGULATION IN THE GENUS MUS RM Bush, K Paigen	Evolution; international journal of organic evolution	1992
Hepatic Storage of Glycosaminoglycans in Feline and Canine Models of Mucopolysaccharidoses I, VI, and VII ME Haskins, EJ Otis, JE Hayden, PF Jezyk, L Stramm	Veterinary pathology	1992
Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer JH Wolfe, MS Sands, JE Barker, B Gwynn, LB Rowe, CA Vogler, EH Birkenmeier	Nature	1992
Mouse Chromosome 5 CA Kozak, DA Stephenson	Mammalian Genome	1992
Mouse map of paralogous genes JH Nadeau, M Kosowsky	Mammalian Genome	1991
Mouse chromosome 5 CA Kozak, DA Stephenson	Mammalian Genome	1991
A murine model of mucopolysaccharidosis VII. Gross and microscopic findings in beta-glucuronidase-deficient mice C Vogler, EH Birkenmeier, WS Sly, B Levy, C Pegors, JW Kyle, WG Beamer	The American Journal of Pathology	1990
Restoration of normal lysosomal function in mucopolysaccharidosis type VII cells by retroviral vector-mediated gene transfer JH Wolfe, EH Schuchman, LE Stramm, EA Concaugh, ME Haskins, GD Aguirre, DF Patterson, RJ Desnick, E Gilboa	Proceedings of the National Academy of Sciences	1990
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene JW Kyle, EH Birkenmeier, B Gwynn, C Vogler, PC Hoppe, JW Hoffmann, WS Sly	Proceedings of the National Academy of Sciences	1990
Nonautologous gene therapy with implantable devices PL Chang	IEEE Engineering in Medicine and Biology Magazine
Analysis of the 5′ flanking region of the human β-glucuronidase gene JM Shipley, RD Miller, BM Wu, JH Grubb, SG Christensen, JW Kyle, WS Sly	Genomics	1991
Cloning and characterization of the human β-glucuronidase gene RD Miller, JW Hoffmann, PP Powell, JW Kyle, JM Shipley, DR Bachinsky, WS Sly	Genomics	1990

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