Cargando…

Enzyme replacement therapy for Farber disease: Proof-of-concept studies in cells and mice

A series of studies were carried out in Farber disease (OMIM #228000) cells and mice to evaluate the feasibility of enzyme replacement therapy (ERT) for this disorder. Media from Chinese hamster ovary (CHO) cells overexpressing human recombinant acid ceramidase (rhAC) was used to treat fibroblasts f...

Descripción completa

Detalles Bibliográficos
Autores principales:	He, Xingxuan, Dworski, Shaalee, Zhu, Changzhi, DeAngelis, Victor, Solyom, Alex, Medin, Jeffrey A., Simonaro, Calogera M., Schuchman, Edward H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2017
Materias:	Regular Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5338723/ https://www.ncbi.nlm.nih.gov/pubmed/28275553 http://dx.doi.org/10.1016/j.bbacli.2017.02.001

Ejemplares similares

Polyarticular arthritis as presenting feature of farber disease: a lysosomal storage disease involving inflammation
por: Sólyom, Alexander, et al.
Publicado: (2014)

Acid Ceramidase Protects Against Hepatic Ischemia/Reperfusion Injury by Modulating Sphingolipid Metabolism and Reducing Inflammation and Oxidative Stress
por: Jiang, Yuan, et al.
Publicado: (2021)

Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI
por: Eliyahu, Efrat, et al.
Publicado: (2011)

Deletion of MCP-1 Impedes Pathogenesis of Acid Ceramidase Deficiency
por: Yu, Fabian P. S., et al.
Publicado: (2018)

Acid Ceramidase Maintains the Chondrogenic Phenotype of Expanded Primary Chondrocytes and Improves the Chondrogenic Differentiation of Bone Marrow-Derived Mesenchymal Stem Cells
por: Simonaro, Calogera M., et al.
Publicado: (2013)

New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy
por: Schuchman, Edward H., et al.
Publicado: (2021)

Acid ceramidase deficiency: Farber disease and SMA-PME
por: Yu, Fabian P. S., et al.
Publicado: (2018)

Dose Responsive Effects of Subcutaneous Pentosan Polysulfate Injection in Mucopolysaccharidosis Type VI Rats and Comparison to Oral Treatment
por: Frohbergh, Michael, et al.
Publicado: (2014)

Pentosan Polysulfate: A Novel Therapy for the Mucopolysaccharidoses
por: Schuchman, Edward H., et al.
Publicado: (2013)

Classic Farber nudes
por: fFarber, Robert
Publicado: (1991)

Systemic ceramide accumulation leads to severe and varied pathological consequences
por: Alayoubi, Abdulfatah M, et al.
Publicado: (2013)

A case of Farber lipogranulomatosis.
por: Kim, Y. J., et al.
Publicado: (1998)

Farber disease: A clinical diagnosis
por: Ekici, Barış, et al.
Publicado: (2012)

Acid Sphingomyelinase Deficiency Ameliorates Farber Disease
por: Beckmann, Nadine, et al.
Publicado: (2019)

Ceramide and Ischemia/Reperfusion Injury
por: He, Xingxuan, et al.
Publicado: (2018)

Identification of a Novel Acid Sphingomyelinase Activity Associated with Recombinant Human Acid Ceramidase
por: He, Xingxuan, et al.
Publicado: (2023)

Safety Study of Sodium Pentosan Polysulfate for Adult Patients with Mucopolysaccharidosis Type II
por: Orii, Kenji, et al.
Publicado: (2019)

Enhanced chondrocyte apoptosis leads to abnormal cartilage in rats and cats with glycosaminoglycan storage diseases
por: Simonaro, CM, et al.
Publicado: (2001)

A new definition of burnout syndrome based on Farber's proposal
por: Montero-Marín, Jesús, et al.
Publicado: (2009)

Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept
por: Cadaoas, Jaclyn, et al.
Publicado: (2020)

Growth Plate Pathology in the Mucopolysaccharidosis Type VI Rat Model—An Experimental and Computational Approach
por: Guevara-Morales, Johana M., et al.
Publicado: (2020)

Farber disease: clinical presentation, pathogenesis and a new approach to treatment
por: Ehlert, Karoline, et al.
Publicado: (2007)

C26-Ceramide as highly sensitive biomarker for the diagnosis of Farber Disease
por: Cozma, Claudia, et al.
Publicado: (2017)

Anaesthetic management of a child with Farber's lipogranulomatosis posted for exploratory laparotomy
por: Choudhary, Nitin, et al.
Publicado: (2019)

On-Tissue Localization of Ceramides and Other Sphingolipids by MALDI Mass Spectrometry Imaging
por: Jones, E. Ellen, et al.
Publicado: (2014)

Actissist: Proof-of-Concept Trial of a Theory-Driven Digital Intervention for Psychosis
por: Bucci, Sandra, et al.
Publicado: (2018)

Recombinant Human Acid Sphingomyelinase as an Adjuvant to Sorafenib Treatment of Experimental Liver Cancer
por: Savić, Radoslav, et al.
Publicado: (2013)

Farber disease: understanding a fatal childhood disorder and dissecting ceramide biology
por: Sands, Mark S
Publicado: (2013)

The Impact of COVID-19 on Clinical Trial Execution at the Dana-Farber Cancer Institute
por: Tolaney, Sara M, et al.
Publicado: (2020)

Pathologic Bladder Microenvironment Attenuates Smooth Muscle Differentiation of Skin Derived Precursor Cells: Implications for Tissue Regeneration
por: Tolg, Cornelia, et al.
Publicado: (2013)

Multidimensional cognitive evaluation of patients with disorders of consciousness using EEG: A proof of concept study
por: Sergent, Claire, et al.
Publicado: (2016)

High field imaging of large-scale neurotransmitter networks: Proof of concept and initial application to epilepsy
por: van Veenendaal, Tamar M., et al.
Publicado: (2018)

Travel burden for patients with multimorbidity – Proof of concept study in a Dutch tertiary care center()
por: Dijkstra, Hidde, et al.
Publicado: (2023)

Intraoperative resection control using arterial spin labeling — Proof of concept, reproducibility of data and initial results
por: Lindner, Thomas, et al.
Publicado: (2017)

Blame-rebalance fMRI neurofeedback in major depressive disorder: A randomised proof-of-concept trial
por: Zahn, Roland, et al.
Publicado: (2019)

Virtual Interviews for the Complex General Surgical Oncology Fellowship: The Dana-Farber/Partners Experience
por: Molina, George, et al.
Publicado: (2020)

Farber disease: report of three cases with joint involvement mimicking juvenile idiopathic arthritis
por: Moghadam, Soheila Hoseinzadeh, et al.
Publicado: (2019)

Mindful walking and cognition in older adults: A proof of concept study using in-lab and ambulatory cognitive measures
por: Yang, Chih-Hsiang, et al.
Publicado: (2021)

Solriamfetol for Excessive Daytime Sleepiness in Parkinson's Disease: Phase 2 Proof‐of‐Concept Trial
por: Videnovic, Aleksandar, et al.
Publicado: (2021)

Farber Disease Mimicking Juvenile Idiopathic Arthritis: The First Reported Case in Qatar and Review of the Literature
por: Al-Naimi, Amal, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_87665ksp8rvdu32t6tulbupii3