JCI - Citations to Intravenous administration of phosphorylated acid alpha-glucosidase leads to uptake of enzyme in heart and skeletal muscle of mice.

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Intravenous administration of phosphorylated acid alpha-glucosidase leads to uptake of enzyme in heart and skeletal muscle of mice.

A T Van der Ploeg, … , N H Brons, A J Reuser

A T Van der Ploeg, … , N H Brons, A J Reuser

Published February 1, 1991
Citation Information: J Clin Invest. 1991;87(2):513-518. https://doi.org/10.1172/JCI115025.

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Citations to this article (57)

Title and authors	Publication	Year
Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases. Puhl AC, Ekins S		2022
Endolysosomal N-glycan processing is critical to attain the most active form of the enzyme acid alpha-glucosidase N Selvan, N Mehta, S Venkateswaran, N Brignol, M Graziano, MO Sheikh, Y McAnany, F Hung, M Madrid, R Krampetz, N Siano, A Mehta, J Brudvig, R Gotschall, JM Weimer, HV Do	The Journal of biological chemistry	2021
Challenges in treating Pompe disease: an industry perspective HV Do, R Khanna, R Gotschall	Annals of translational medicine	2019
AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice BM Doyle, SM Turner, MD Sunshine, PA Doerfler, AE Poirier, LA Vaught, ML Jorgensen, DJ Falk, BJ Byrne, DD Fuller	Molecular Therapy — Methods & Clinical Development	2019
Using human Pompe disease-induced pluripotent stem cell-derived neural cells to identify compounds with therapeutic potential HP Huang, W Chiang, L Stone, CK Kang, CY Chuang, HC Kuo	Human Molecular Genetics	2019
Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease L Harlaar, JY Hogrel, B Perniconi, ME Kruijshaar, D Rizopoulos, N Taouagh, A Canal, E Brusse, PA van Doorn, AT van der Ploeg, P Laforêt, NA van der Beek	Neurology	2019
Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy R Parini, PD Lorenzo, A Dardis, A Burlina, A Cassio, P Cavarzere, D Concolino, RD Casa, F Deodato, MA Donati, A Fiumara, S Gasperini, F Menni, V Pagliardini, M Sacchini, M Spada, R Taurisano, MG Valsecchi, MD Rocco, B Bembi	Orphanet Journal of Rare Diseases	2018
Low Prevalence Estimates of Late-Onset Glycogen Storage Disease Type II in French-Speaking Belgium are not Due to Missed Diagnoses G Remiche, Z Lukacs, DC Kasper, M Abramowicz, M Pandolfo	Journal of neuromuscular diseases	2018
The impact of Pompe disease on smooth muscle: a review AL MCCALL, J SALEMI, P BHANAP, LM STRICKLAND, MK ELMALLAH	Journal of Smooth Muscle Research	2018
Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study CM van Gelder, E Poelman, I Plug, M Hoogeveen-Westerveld, NA van der Beek, AJ Reuser, AT van der Ploeg	Journal of Inherited Metabolic Disease	2016
Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice DJ Falk, MS Soustek, AG Todd, CS Mah, DA Cloutier, JS Kelley, N Clement, DD Fuller, BJ Byrne	Molecular Therapy — Methods & Clinical Development	2015
Diagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert group FA Jasmi, MA Jumah, F Alqarni, N Al-Sannaa, F Al-Sharif, S Bohlega, EJ Cupler, W Fathalla, MA Hamdan, N Makhseed, S Nafissi, Y Nilipour, L Selim, N Shembesh, R Sunbul, SH Tonekaboni	BMC neurology	2015
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV- GAA ) Gene Vector in Patients with Pompe Disease BJ Byrne, B Smith, C Mah, LA Lawson, S Islam, M Corti, D Martin, N Dobija, MV Irwin, T Conlon, B Cleaver, N Clement, SW Collins	Human Gene Therapy Clinical Development	2014
Expression of a secretory α-glucosidase II from Apis cerana indica in Pichia pastoris and its characterization J Kaewmuangmoon, M Kilaso, U Leartsakulpanich, K Kimura, A Kimura, C Chanchao	BMC Biotechnology	2013
Production of a Functional Human Acid Maltase in Tobacco Seeds: Biochemical Analysis, Uptake by Human GSDII Cells, and In Vivo Studies in GAA Knockout Mice F Martiniuk, S Reggi, KM Tchou-Wong, WN Rom, M Busconi, C Fogher	Applied Biochemistry and Biotechnology	2013
Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in pompe mice JA Maga, J Zhou, R Kambampati, S Peng, X Wang, RN Bohnsack, A Thomm, S Golata, P Tom, NM Dahms, BJ Byrne, JH LeBowitz	The Journal of biological chemistry	2012
Adamantyl Glycosphingolipids Provide a New Approach to the Selective Regulation of Cellular Glycosphingolipid Metabolism M Kamani, M Mylvaganam, R Tian, B Rigat, B Binnington, C Lingwood	The Journal of biological chemistry	2011
Murine muscle cell models for Pompe disease and their use in studying therapeutic approaches S Takikita, R Myerowitz, K Zaal, N Raben, PH Plotz	Molecular Genetics and Metabolism	2009
Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease M Beck	Therapeutics and Clinical Risk Management	2009
The Significance of Glycosylation Analysis in Development of Biopharmaceuticals N Kawasaki, S Itoh, N Hashii, D Takakura, Y Qin, X Huang, T Yamaguchi	Biological & Pharmaceutical Bulletin	2009
Pompe's disease AT van der Ploeg, AJ Reuser	The Lancet	2008
Ability of Adeno-Associated Virus Serotype 8-Mediated Hepatic Expression of Acid α-Glucosidase to Correct the Biochemical and Motor Function Deficits of Presymptomatic and Symptomatic Pompe Mice RJ Ziegler, SD Bercury, J Fidler, MA Zhao, J Foley, TV Taksir, S Ryan, BL Hodges, RK Scheule, LS Shihabuddin, SH Cheng	Human Gene Therapy	2008
Current Strategies in the Management of Lysosomal Storage Diseases BA Heese	Seminars in Pediatric Neurology	2008
Pompe disease: Current state of treatment modalities and animal models TM Geel, PM McLaughlin, LF Leij, MH Ruiters, KE Niezen-Koning	Molecular Genetics and Metabolism	2007
Alglucosidase alfa: first available treatment for Pompe disease M Nicolino	Therapy	2007
Lysosomal Storage Disorders JA Barranger, MA Cabrera-Salazar		2007
Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease T Fukuda, L Ewan, M Bauer, RJ Mattaliano, K Zaal, E Ralston, PH Plotz, N Raben	Annals of Neurology	2006
New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy M Beck	Human Genetics	2006
Hyaluronidase increases the biodistribution of acid α-1,4 glucosidase in the muscle of Pompe disease mice: An approach to enhance the efficacy of enzyme replacement therapy R Matalon, S Surendran, GA Campbell, K Michals-Matalon, SK Tyring, J Grady, S Cheng, E Kaye	Biochemical and Biophysical Research Communications	2006
Replacing acid α-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers N Raben, T Fukuda, AL Gilbert, D Jong, BL Thurberg, RJ Mattaliano, P Meikle, JJ Hopwood, K Nagashima, K Nagaraju, PH Plotz	Molecular Therapy	2005
Conjugation of Mannose 6-Phosphate-containing Oligosaccharides to Acid α-Glucosidase Improves the Clearance of Glycogen in Pompe Mice Y Zhu, X Li, J Kyazike, Q Zhou, BL Thurberg, N Raben, RJ Mattaliano, SH Cheng	The Journal of biological chemistry	2004
Lipoprotein Receptor Binding, Cellular Uptake, and Lysosomal Delivery of Fusions between the Receptor-associated Protein (RAP) and α-l-Iduronidase or Acid α-Glucosidase WS Prince, LM McCormick, DJ Wendt, PA Fitzpatrick, KL Schwartz, AI Aguilera, V Koppaka, TM Christianson, MC Vellard, N Pavloff, JF Lemontt, M Qin, CM Starr, G Bu, TC Zankel	The Journal of biological chemistry	2004
Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model F Xu, E Ding, SX Liao, F Migone, J Dai, A Schneider, D Serra, YT Chen, A Amalfitano	Gene Therapy	2004
Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors TJ Fraites, MR Schleissing, RA Shanely, GA Walter, DA Cloutier, I Zolotukhin, DF Pauly, N Raben, PH Plotz, SK Powers, PD Kessler, BJ Byrne	Molecular Therapy	2002
Helios Gene Gun Particle Delivery for Therapy of Acid Maltase Deficiency F Martiniuk, A Chen, A Mack, V Donnabella, A Slonim, L Bulone, E Arvanitopoulos, N Raben, P Plotz, WN Rom	DNA and Cell Biology	2002
Enzyme therapy for Pompe disease: from science to industrial enterprise AJ Reuser, H Hout, AG Bijvoet, MA Kroos, MP Verbeet, AT Ploeg	European Journal of Pediatrics	2002
Intercellular Transfer of the Virally Derived Precursor Form of Acid α-Glucosidase Corrects the Enzyme Deficiency in Inherited Cardioskeletal Myopathy Pompe Disease DF Pauly, TJ Fraites, C Toma, HS Bayes, ML Huie, R Hirschhorn, PH Plotz, N Raben, PD Kessler, BJ Byrne	Human Gene Therapy	2001
Approach to Gene Therapy of Glycogenosis Type II (Pompe Disease) L Poenaru	Molecular Genetics and Metabolism	2000
Correction of Glycogen Storage Disease Type II by Enzyme Replacement with a Recombinant Human Acid Maltase Produced by Over-Expression in a CHO-DHFRneg Cell Line F Martiniuk, A Chen, V Donnabella, E Arvanitopoulos, AE Slonim, N Raben, P Plotz, WN Rom	Biochemical and Biophysical Research Communications	2000
Towards a molecular therapy for glycogen storage disease type II (Pompe disease) YT Chen, A Amalfitano	Molecular Medicine Today	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
Allosteric Activation of Acid α-Glucosidase by the Human Papillomavirus E7 Protein W Zwerschke, B Mannhardt, P Massimi, S Nauenburg, D Pim, W Nickel, L Banks, AJ Reuser, P Jansen-Dürr	The Journal of biological chemistry	2000
The Molecular Background of Glycogen Metabolism Disorders ON Elpeleg	Journal of Pediatric Endocrinology and Metabolism	1999
Human acid alpha-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II AG Bijvoet, HV Hirtum, MA Kroos, EH van de Kamp, O Schoneveld, P Visser, JP Brakenhoff, M Weggeman, EJ van Corven, AT van der Ploeg, AJ Reuser	Human Molecular Genetics	1999
Recombinant Human Acid α-Glucosidase Corrects Acidα-Glucosidase-Deficient Human Fibroblasts, Quail Fibroblasts, and Quail Myoblasts HW Yang, T Kikuchi, Y Hagiwara, M Mizutani, YT Chen, JL van Hove	Pediatric Research	1998
Adenovirus-Mediated Transfer of the Acid -Glucosidase Gene into Fibroblasts, Myoblasts and Myotubes from Patients with Glycogen Storage Disease Type II Leads to High Level Expression of Enzyme and Corrects Glycogen Accumulation MP Nicolino, JP Puech, EJ Kremer, AJ Reuser, C Mbebi, M Verdiere-Sahuque, A Kahn, L Poenaru	Human Molecular Genetics	1998
Targeted Disruption of the Acid α-Glucosidase Gene in Mice Causes an Illness with Critical Features of Both Infantile and Adult Human Glycogen Storage Disease Type II N Raben, K Nagaraju, E Lee, P Kessler, B Byrne, L Lee, M LaMarca, C King, J Ward, B Sauer, P Plotz	The Journal of biological chemistry	1998
Retroviral Transfer of Acidα-Glucosidase cDNA to Enzyme-Deficient Myoblasts Results in Phenotypic Spread of the Genotypic Correction by Both Secretion and Fusion JZ Zaretsky, F Candotti, C Boerkoel, EM Adams, JW Yewdell, RM Blaese, PH Plotz	Human Gene Therapy	1997
Skeletal Muscle Weakness and Dysphagia Caused by Acid Maltase Deficiency: Nutritional Consequences of Coincident Celiac Sprue TS King, JR Anderson, EP Wraight, JO Hunter, TM Cox	JPEN. Journal of parenteral and enteral nutrition	1997
Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome AC Crawley, DA Brooks, VJ Muller, BA Petersen, EL Isaac, J Bielicki, BM King, CD Boulter, AJ Moore, NL Fazzalari, DS Anson, S Byers, JJ Hopwood	Journal of Clinical Investigation	1996
High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease JL van Hove, HW Yang, JY Wu, RO Brady, YT Chen	Proceedings of the National Academy of Sciences	1996
Expression of cDNA-encoded human acid α-glucosidase in milk of transgenic mice AG Bijvoet, MA Kroos, FR Pieper, HA de Boer, AJ Reuser, AT van der Ploeg, MP Verbeet	Biochimica et Biophysica Acta (BBA) - Gene Structure and Expression	1996
Isolation and Characterisation of a Recombinant, Precursor form of Lysosomal Acid alpha-Glucosidase M Fuller, AD Ploeg, AJ Reuser, DS Anson, JJ Hopwood	European Journal of Biochemistry	1995
Glycogenosis type II (acid maltase deficiency) AJ Reuser, MA Kroos, MM Hermans, AG Bijvoet, MP Verbeet, OP van Diggelen, WJ Kleijer, AT van der Ploeg	Muscle & Nerve	1995
Recombinant Human Acid α-Glucosidase Generated in Bacteria: Antigenic, but Enzymatically Inactive F Martiniuk, S Tzall, A Chen	DNA and Cell Biology	1992
Long-term effects of bone marrow transplantation on lysosomal enzyme replacement in β-glucuronidase-deficient mice G Bou-Gharios, G Adams, P Pace, I Olsen	Journal of Inherited Metabolic Disease	1992
Metabolic cardiomyopathies ME Tripp	Progress in Pediatric Cardiology	1992

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