Cargando…

The Pharmacological Chaperone N-butyldeoxynojirimycin Enhances Enzyme Replacement Therapy in Pompe Disease Fibroblasts

In spite of the progress in the treatment of lysosomal storage diseases (LSDs), in some of these disorders the available therapies show limited efficacy and a need exists to identify novel therapeutic strategies. We studied the combination of enzyme replacement and enzyme enhancement by pharmacologi...

Descripción completa

Detalles Bibliográficos
Autores principales:	Porto, Caterina, Cardone, Monica, Fontana, Federica, Rossi, Barbara, Tuzzi, Maria Rosaria, Tarallo, Antonietta, Barone, Maria Vittoria, Andria, Generoso, Parenti, Giancarlo
Formato:	Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2009
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2835191/ https://www.ncbi.nlm.nih.gov/pubmed/19293774 http://dx.doi.org/10.1038/mt.2009.53

Ejemplares similares

Abnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts
por: Cardone, Monica, et al.
Publicado: (2008)

S2.2 Enzyme replacement therapy in the infantile-onset Pompe disease
por: Parenti, Giancarlo, et al.
Publicado: (2011)

S1.2 The pediatrician and the early onset Pompe disease
por: Fecarotta, Simona, et al.
Publicado: (2011)

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease
por: Tarallo, Antonietta, et al.
Publicado: (2021)

Improvement of dysphagia in a child affected by Pompe disease treated with enzyme replacement therapy
por: Fecarotta, Simona, et al.
Publicado: (2013)

Carnitine is a pharmacological allosteric chaperone of the human lysosomal α-glucosidase
por: Iacono, Roberta, et al.
Publicado: (2021)

Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe disease
por: Schaaf, Gerben J., et al.
Publicado: (2018)

N -butyldeoxynojirimycin reduces growth and ganglioside content of experimental mouse brain tumours
por: Ranes, M K, et al.
Publicado: (2001)

Aortopathies in mouse models of Pompe, Fabry and Mucopolysaccharidosis IIIB lysosomal storage diseases
por: Belfiore, Maria Paola, et al.
Publicado: (2020)

Pharmacological Chaperone Therapy for Pompe Disease
por: Borie-Guichot, Marc, et al.
Publicado: (2021)

Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease
por: Spampanato, Carmine, et al.
Publicado: (2013)

AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease
por: Gatto, Francesca, et al.
Publicado: (2017)

Respiratory manifestations in patients with inherited metabolic diseases
por: Santamaria, Francesca, et al.
Publicado: (2013)

Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics
por: Parenti, Giancarlo
Publicado: (2009)

Enzyme replacement therapy (ERT) in pompe disease
por: Fiumara, Agata
Publicado: (2014)

Genetic counseling in Pompe disease
por: Taglia, Antonella, et al.
Publicado: (2011)

Pathogenesis of Mucopolysaccharidoses, an Update
por: Fecarotta, Simona, et al.
Publicado: (2020)

S2.5 The nutritional approach to Pompe disease
por: Esposito, Katia, et al.
Publicado: (2011)

Structure of human lysosomal acid α-glucosidase–a guide for the treatment of Pompe disease
por: Roig-Zamboni, Véronique, et al.
Publicado: (2017)

Enzyme replacement therapy and fatigue in adults with Pompe disease
por: Güngör, Deniz, et al.
Publicado: (2013)

Involvement of endocrine system in a patient affected by Glycogen storage disease 1b: speculation on the role of autoimmunity
por: Melis, Daniela, et al.
Publicado: (2014)

Impact of enzyme replacement therapy on survival in adults with Pompe disease
por: Güngör, Deniz, et al.
Publicado: (2013)

Enzyme replacement therapy and antibodies in late-onset Pompe disease
por: Schneider, I., et al.
Publicado: (2014)

Abstract 2189: The Impact of N-butyldeoxynojirimycin and 1-deoxymannojirimycin on SARS-CoV-2 S1 Subunit and Angiotensin-Converting Enzyme 2 Interaction in Intestinal Epithelial Cells
por: El Khoury, Marianne, et al.
Publicado: (2023)

Impact in Italy of research in pediatrics
por: Andria, Generoso
Publicado: (2014)

Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel
por: Gragnaniello, Vincenza, et al.
Publicado: (2022)

Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease
por: Cousens, Leslie P., et al.
Publicado: (2012)

Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease
por: Meena, Naresh Kumar, et al.
Publicado: (2020)

The impact of interrupting enzyme replacement therapy in late-onset Pompe disease
por: Wenninger, Stephan, et al.
Publicado: (2021)

Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients
por: van der Meijden, Jan C., et al.
Publicado: (2018)

Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease()
por: Chien, Yin-Hsiu, et al.
Publicado: (2017)

Standardized nursing management of enzyme replacement therapy for late-onset Pompe disease
por: Tang, Shan, et al.
Publicado: (2021)

Different Approaches to Analyze Muscle Fat Replacement With Dixon MRI in Pompe Disease
por: Alonso-Jiménez, Alicia, et al.
Publicado: (2021)

Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease
por: Fiege, Lina, et al.
Publicado: (2023)

Pompes turbomoleculaires
por: Versolatto, B
Publicado: (1982)

Postbiotic Preparation of Lacticaseibacillus rhamnosus GG against Diarrhea and Oxidative Stress Induced by Spike Protein of SARS-CoV-2 in Human Enterocytes
por: Poeta, Marco, et al.
Publicado: (2023)

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
por: Kanters, Tim A., et al.
Publicado: (2017)

Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease
por: Cheon, Chong Kun
Publicado: (2019)

Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy
por: Hsueh, Chien-Yu, et al.
Publicado: (2021)

Characteristics of Pompe disease in China: a report from the Pompe registry
por: Zhao, Yuying, et al.
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_e3matte6s3ihrvsbe9bjetov8p