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Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.

L H O'Connor, … , B Levy, M S Sands

L H O'Connor, … , B Levy, M S Sands

Published April 1, 1998
Citation Information: J Clin Invest. 1998;101(7):1394-1400. https://doi.org/10.1172/JCI1773.

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Citations to this article (90)

Title and authors	Publication	Year
Enzyme therapy: a forerunner in catalyzing a healthy society? S Datta, KN Rajnish, CG Doss, SM Samuel, E Selvarajan, H Zayed	Expert Opinion on Biological Therapy	2020
Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII J Cadaoas, G Boyle, S Jungles, S Cullen, M Vellard, JH Grubb, A Jurecka, W Sly, E Kakkis	Molecular Genetics and Metabolism	2020
Hearing Loss in Mucopolysaccharidoses: Current Knowledge and Future Directions J Wolfberg, K Chintalapati, S Tomatsu, K Nagao	Diagnostics	2020
Vestronidase Alfa: A Review in Mucopolysaccharidosis VII EH McCafferty, LJ Scott	BioDrugs	2019
A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease P Harmatz, CB Whitley, RY Wang, M Bauer, W Song, C Haller, E Kakkis	Molecular Genetics and Metabolism	2018
Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials Y Qi, K McKeever, J Taylor, C Haller, W Song, SA Jones, J Shi	Clinical Pharmacokinetics	2018
Emerging drugs for the treatment of mucopolysaccharidoses R Giugliani, A Federhen, F Vairo, C Vanzella, G Pasqualim, LM da Silva, L Giugliani, AP de Boer, CF de Souza, U Matte, G Baldo	Expert Opinion on Emerging Drugs	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
Bilateral single-site intracerebral injection of a nonpathogenic herpes simplex virus-1 vector decreases anxiogenic behavior in MPS VII mice W Liu, G Griffin, T Clarke, MK Parente, RJ Valentino, JH Wolfe, NW Fraser	Molecular Therapy — Methods & Clinical Development	2015
First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient JE Fox, L Volpe, J Bullaro, ED Kakkis, WS Sly	Molecular Genetics and Metabolism	2015
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII BL Gurda, AD de Lataillade, P Bell, Y Zhu, H Yu, P Wang, J Bagel, CH Vite, T Sikora, C Hinderer, R Calcedo, AD Yox, RA Steet, T Ruane, P O'Donnell, G Gao, JM Wilson, M Casal, KP Ponder, ME Haskins	Molecular Therapy	2015
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
Assessment of hearing loss by pure-tone audiometry in patients with mucopolysaccharidoses HY Lin, SC Shih, CK Chuang, KS Lee, MR Chen, HC Lin, PC Chiu, DM Niu, SP Lin	Molecular Genetics and Metabolism	2014
Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice G Baldo, FQ Mayer, BZ Martinelli, TG de Carvalho, FS Meyer, PG de Oliveira, L Meurer, Â Tavares, U Matte, R Giugliani	Molecular Genetics and Metabolism	2013
Impaired critical period plasticity in primary auditory cortex of fragile X model mice H Kim, R Gibboni, C Kirkhart, S Bao	The Journal of neuroscience : the official journal of the Society for Neuroscience	2013
Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease YH Chen, K Claflin, JC Geoghegan, BL Davidson	Molecular Therapy	2012
Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII HT Huynh, JH Grubb, C Vogler, WS Sly	Proceedings of the National Academy of Sciences	2012
Examination of intravenous and intra-CSF protein delivery for treatment of neurological disease KM Hemsley, AJ Luck, AC Crawley, S Hassiotis, H Beard, B King, T Rozek, T Rozaklis, M Fuller, JJ Hopwood	European Journal of Neuroscience	2009
Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome D Pan, AS 2nd, CV Vorhees, MT Williams	Brain Research	2008
Adeno-associated Virus Type 5 Reduces Learning Deficits and Restores Glutamate Receptor Subunit Levels in MPS VII Mice CNS G Liu, YH Chen, X He, I Martins, JA Heth, JA Chiorini, BL Davidson	Molecular Therapy	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
Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA AL Roberts, MH Rees, S Klebe, JM Fletcher, S Byers	Molecular Genetics and Metabolism	2007
Lysosomal Storage Disorders JA Barranger, MA Cabrera-Salazar		2007
Reversibility of cellular and organ pathology in enzyme replacement trials in animal models of lysosomal storage diseases V Gieselmann	Acta Paediatrica	2006
Histopathological and behavioral improvement of murine mucopolysaccharidosis type VII by intracerebral transplantation of neural stem cells Y Fukuhara, XK Li, Y Kitazawa, M Inagaki, K Matsuoka, M Kosuga, R Kosaki, T Shimazaki, H Endo, A Umezawa, H Okano, T Takahashi, T Okuyama	Molecular Therapy	2005
AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease MA Passini, SL Macauley, MR Huff, TV Taksir, J Bu, IH Wu, PA Piepenhagen, JC Dodge, LS Shihabuddin, CR O'Riordan, EH Schuchman, GR Stewart	Molecular Therapy	2005
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
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
International Review of Neurobiology ZT Bloomgarden	International review of neurobiology	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
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
Enzyme-Replacement Therapy from Birth Delays the Development of Behavior and Learning Problems in Mucopolysaccharidosis Type IIIA Mice BL Gliddon, JJ Hopwood	Pediatric Research	2004
Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human α-l-iduronidase gene SD Hartung, JL Frandsen, D Pan, BL Koniar, P Graupman, R Gunther, WC Low, CB Whitley, RS McIvor	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
Targeting macrophages with baculovirus‐produced lysosomal enzymes: implications for enzyme replacement therapy of the glycoprotein storage disorder galactosialidosis EJ Bonten, D Wang, JN Toy, L Mann, A Mignardot, G Yogalingam, A d'Azzo	The FASEB Journal	2004
Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I R Kakavanos, CT Turner, JJ Hopwood, ED Kakkis, DA Brooks	The Lancet	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
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
Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer N Takaura, T Yagi, M Maeda, E Nanba, A Oshima, Y Suzuki, T Yamano, A Tanaka	Gene Therapy	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
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
Enzyme replacement therapy: from concept to clinical practice WS Sly	Acta Paediatrica	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
Inner ear pathology in the mucopolysaccharidosis VII mouse KK Ohlemiller, AK Hennig, JM Lett, AF Heidbreder, MS Sands	Hearing Research	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
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
Mucopolysaccharidosis Type VII (Sly Syndrome) Presenting as Neonatal Cholestasis With Hepatosplenomegaly PM Gillett, RA Schreiber, GP Jevon, DM Israel, T Warshawski, H Vallance, LA Clarke	Journal of Pediatric Gastroenterology and Nutrition	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
Applications of the Morris water maze in the study of learning and memory R D'Hooge, PP de Deyn	Brain research. Brain research reviews	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
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice A Consiglio, A Quattrini, S Martino, JC Bensadoun, D Dolcetta, A Trojani, G Benaglia, S Marchesini, V Cestari, A Oliverio, C Bordignon, L Naldini	Nature Medicine	2001
The HIV Tat protein transduction domain improves the biodistribution of β-glucuronidase expressed from recombinant viral vectors H Xia, Q Mao, BL Davidson	Nature Biotechnology	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
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
Enzyme Replacement Therapy in Mucopolysaccharidosis I: Altered Distribution and Targeting of α-l-Iduronidase in Immunized Rats CT Turner, JJ Hopwood, DA Brooks	Molecular Genetics and Metabolism	2000
Abnormal Immune Function In Vivo in a Murine Model of Lysosomal Storage Disease TM Daly, RG Lorenz, MS Sands	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
Enzyme replacement therapy in a mouse model of aspartylglycosaminuria U Dunder, V Kaartinen, P Valtonen, E Väänänen, VM Kosma, N Heisterkamp, J Groffen, I Mononen	The FASEB Journal	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
Phenotype Correction in Murine Mucopolysaccharidosis Type VII by Transplantation of Human Amniotic Epithelial Cells after Adenovirus-Mediated Gene Transfer M Kosuga, S Takahashi, K Sasaki, S Enosawa, XK Li, S Okuyama, M Fujino, S Suzuki, M Yamada, N Matsuo, N Sakuragawa, T Okuyama	Cell Transplantation	2000
Infusion of recombinant human acid sphingomyelinase into Niemann-Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology SI Miranda, XI He, CM Simonaro, S Gatt, A Dagan, RJ Desnick, EH Schuchman	The FASEB Journal	2000
Caprine Mucopolysaccharidosis IIID: A Preliminary Trial of Enzyme Replacement Therapy E Downs-Kelly, MZ Jones, J Alroy, KT Cavanagh, B King, RE Lucas, JC Baker, SA Kraemer, JJ Hopwood	Journal of Molecular Neuroscience	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
Correction of Enzymatic and Lysosomal Storage Defects in Fabry Mice by Adenovirus-Mediated Gene Transfer RJ Ziegler, NS Yew, C Li, M Cherry, P Berthelette, H Romanczuk, YA Ioannou, KM Zeidner, RJ Desnick, SH Cheng	Human Gene Therapy	1999
Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease TM Daly, C Vogler, B Levy, ME Haskins, MS Sands	Proceedings of the National Academy of Sciences	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
Is There Treatment for “Genetic” Disease? CR Scriver, EP Treacy	Molecular Genetics and Metabolism	1999
Human Glycosylation Disorders and Sugar Supplement Therapy HH Freeze	Biochemical and Biophysical Research Communications	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
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