Cargando…

Mannose receptor-mediated delivery of moss-made α-galactosidase A efficiently corrects enzyme deficiency in Fabry mice

Enzyme replacement therapy (ERT) is an effective treatment for several lysosomal storage disorders (LSDs). Intravenously infused enzymes are taken up by tissues through either the mannose 6-phosphate receptor (M6PR) or the mannose receptor (MR). It is generally believed that M6PR-mediated endocytosi...

Descripción completa

Detalles Bibliográficos
Autores principales:	Shen, Jin-Song, Busch, Andreas, Day, Taniqua S., Meng, Xing-Li, Yu, Chun I., Dabrowska-Schlepp, Paulina, Fode, Benjamin, Niederkrüger, Holger, Forni, Sabrina, Chen, Shuyuan, Schiffmann, Raphael, Frischmuth, Thomas, Schaaf, Andreas
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer Netherlands 2015
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4754329/ https://www.ncbi.nlm.nih.gov/pubmed/26310963 http://dx.doi.org/10.1007/s10545-015-9886-9

Ejemplares similares

Comparison of efficacy between subcutaneous and intravenous application of moss‐aGal in the mouse model of Fabry disease
por: Dabrowska‐Schlepp, Paulina, et al.
Publicado: (2023)

Uptake of moss‐derived human recombinant GAA in Gaa (−/−) mice
por: Hintze, Stefan, et al.
Publicado: (2021)

Moss-Derived Human Recombinant GAA Provides an Optimized Enzyme Uptake in Differentiated Human Muscle Cells of Pompe Disease
por: Hintze, Stefan, et al.
Publicado: (2020)

Optimizing human α-galactosidase for treatment of Fabry disease
por: Hallows, William C., et al.
Publicado: (2023)

Dysregulated DNA methylation in the pathogenesis of Fabry disease
por: Shen, Jin-Song, et al.
Publicado: (2022)

Adipocytes participate in storage in α-galactosidase deficiency (Fabry disease)
por: Hůlková, Helena, et al.
Publicado: (2010)

A Pharmacogenetic Approach to Identify Mutant Forms of α-Galactosidase A that Respond to a Pharmacological Chaperone for Fabry Disease
por: Wu, Xiaoyang, et al.
Publicado: (2011)

Moss‐made pharmaceuticals: from bench to bedside
por: Reski, Ralf, et al.
Publicado: (2015)

Receptor-Mediated Endocytosis of α-Galactosidase A in Human Podocytes in Fabry Disease
por: Prabakaran, Thaneas, et al.
Publicado: (2011)

Plasma mutant α-galactosidase A protein and globotriaosylsphingosine level in Fabry disease
por: Tsukimura, Takahiro, et al.
Publicado: (2014)

A Novel α-Galactosidase A Splicing Mutation Predisposes to Fabry Disease
por: Li, Ping, et al.
Publicado: (2019)

Rats deficient in α-galactosidase A develop ocular manifestations of Fabry disease
por: Miller, James J., et al.
Publicado: (2019)

Globotriaosylsphingosine Accumulation and Not Alpha-Galactosidase-A Deficiency Causes Endothelial Dysfunction in Fabry Disease
por: Namdar, Mehdi, et al.
Publicado: (2012)

A Novel Frameshift Mutation of Galactosidase-alpha in Fabry Disease Restricted to Dermatologic Manifestations
por: Kim, Dae Hun, et al.
Publicado: (2013)

Affective and cognitive behavior in the alpha-galactosidase A deficient mouse model of Fabry disease
por: Hofmann, Lukas, et al.
Publicado: (2017)

Mannose 6-Phosphate Receptor and Sortilin Mediated Endocytosis of α-Galactosidase A in Kidney Endothelial Cells
por: Prabakaran, Thaneas, et al.
Publicado: (2012)

Correction: Globotriaosylceramide Accumulation and Not Alpha-Galactosidase-A Deficiency Causes Endothelial Dysfunction in Fabry Disease
por: Namdar, Mehdi, et al.
Publicado: (2012)

Alpha-Galactosidase A p.A143T, a non-Fabry disease-causing variant
por: Lenders, Malte, et al.
Publicado: (2016)

Human α-Galactosidase A Mutants: Priceless Tools to Develop Novel Therapies for Fabry Disease
por: Modrego, Andrea, et al.
Publicado: (2021)

α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice
por: Rodríguez-Castejón, Julen, et al.
Publicado: (2021)

Curcumin Has Beneficial Effects on Lysosomal Alpha-Galactosidase: Potential Implications for the Cure of Fabry Disease
por: Monticelli, Maria, et al.
Publicado: (2023)

Identification of a Missense Mutation in the α-galactosidase A Gene in a Chinese Family with Fabry Disease
por: Wu, Yuan, et al.
Publicado: (2018)

The New Pharmacological Chaperones PBXs Increase α-Galactosidase A Activity in Fabry Disease Cellular Models
por: Besada, Pedro, et al.
Publicado: (2021)

Drug Repositioning for Fabry Disease: Acetylsalicylic Acid Potentiates the Stabilization of Lysosomal Alpha-Galactosidase by Pharmacological Chaperones
por: Monticelli, Maria, et al.
Publicado: (2022)

Overcoming establishment thresholds for peat mosses in human‐made bog pools
por: Temmink, Ralph J. M., et al.
Publicado: (2021)

Replacement of α-galactosidase A in Fabry disease: effect on fibroblast cultures compared with biopsied tissues of treated patients
por: Keslová-Veselíková, Jana, et al.
Publicado: (2008)

Functional Characterisation of Alpha-Galactosidase A Mutations as a Basis for a New Classification System in Fabry Disease
por: Lukas, Jan, et al.
Publicado: (2013)

Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A
por: Biferi, Maria Grazia, et al.
Publicado: (2020)

α-Galactosidase a Deficiency in Fabry Disease Leads to Extensive Dysregulated Cellular Signaling Pathways in Human Podocytes
por: Jehn, Ulrich, et al.
Publicado: (2021)

Reduced α-galactosidase A activity in zebrafish (Danio rerio) mirrors distinct features of Fabry nephropathy phenotype
por: Elsaid, Hassan O.A., et al.
Publicado: (2022)

Novel α-galactosidase A gene mutation in a Chinese Fabry disease family: A case report
por: Fu, An-Yi, et al.
Publicado: (2022)

Use of endogenous signal sequences for transient production and efficient secretion by moss (Physcomitrella patens) cells
por: Schaaf, Andreas, et al.
Publicado: (2005)

Composition and structure of photosystem I in the moss Physcomitrella patens
por: Busch, Andreas, et al.
Publicado: (2013)

Distributions of Globotriaosylceramide Isoforms, and Globotriaosylsphingosine and Its Analogues in an α-Galactosidase A Knockout Mouse, a Model of Fabry Disease
por: Sueoka, Hideaki, et al.
Publicado: (2015)

Rapid Immunochromatographic Detection of Serum Anti-α-Galactosidase A Antibodies in Fabry Patients after Enzyme Replacement Therapy
por: Nakano, Sachie, et al.
Publicado: (2015)

Identification of an Allosteric Binding Site on Human Lysosomal Alpha-Galactosidase Opens the Way to New Pharmacological Chaperones for Fabry Disease
por: Citro, Valentina, et al.
Publicado: (2016)

Human Alpha Galactosidases Transiently Produced in Nicotiana benthamiana Leaves: New Insights in Substrate Specificities with Relevance for Fabry Disease
por: Kytidou, Kassiani, et al.
Publicado: (2017)

Predicting the Development of Anti-Drug Antibodies against Recombinant alpha-Galactosidase A in Male Patients with Classical Fabry Disease
por: van der Veen, Sanne J., et al.
Publicado: (2020)

Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice
por: Xu, Su, et al.
Publicado: (2015)

Inhibition of Arachidonate 12/15-Lipoxygenase Improves α-Galactosidase Efficacy in iPSC-Derived Cardiomyocytes from Fabry Patients
por: Chien, Yueh, et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_cfrt4g46flrmh413gu2mgqkbuf