Cargando…

Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy

Mucopolysaccharidosis type II (MPSII), or Hunter syndrome, is a devastating disorder associated with a shortened life expectancy. Patients affected by MPSII have a variety of symptoms that affect all organs of the body and may include progressive cognitive impairment. MPSII is due to inactivity of t...

Descripción completa

Detalles Bibliográficos
Autores principales:	Polito, Vinicia Assunta, Abbondante, Serena, Polishchuk, Roman S., Nusco, Edoardo, Salvia, Rosaria, Cosma, Maria Pia
Formato:	Texto
Lenguaje:	English
Publicado:	Oxford University Press 2010
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2989894/ https://www.ncbi.nlm.nih.gov/pubmed/20876612 http://dx.doi.org/10.1093/hmg/ddq420

Ejemplares similares

Glial degeneration with oxidative damage drives neuronal demise in MPSII disease
por: Zalfa, Cristina, et al.
Publicado: (2016)

Targeting Brain Disease in MPSII: Preclinical Evaluation of IDS-Loaded PLGA Nanoparticles
por: Rigon, Laura, et al.
Publicado: (2019)

Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease
por: Alliegro, Marialuisa, et al.
Publicado: (2016)

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

Case report of endoprosthesis -Y implantation in severe respiratory failure in the MPSII patient; comparison with literature data
por: Gocyk, Wojciech, et al.
Publicado: (2020)

Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II
por: Cho, Sung Yoon, et al.
Publicado: (2015)

Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy
por: Ellison, Stuart, et al.
Publicado: (2023)

Vascular and parenchymal amyloid pathology in an Alzheimer disease knock-in mouse model: interplay with cerebral blood flow
por: Li, Hongmei, et al.
Publicado: (2014)

Modulation of microRNA editing, expression and processing by ADAR2 deaminase in glioblastoma
por: Tomaselli, Sara, et al.
Publicado: (2015)

Impaired parkin-mediated mitochondrial targeting to autophagosomes differentially contributes to tissue pathology in lysosomal storage diseases
por: de Pablo-Latorre, Raquel, et al.
Publicado: (2012)

Induction of Autophagy Promotes Clearance of RHO(P23H) Aggregates and Protects From Retinal Degeneration
por: Intartaglia, Daniela, et al.
Publicado: (2022)

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)

Preclinical Enzyme Replacement Therapy with a Recombinant β-Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis
por: Weesner, Jason Andrew, et al.
Publicado: (2022)

Retinal Degeneration in MPS-IIIA Mouse Model
por: Intartaglia, Daniela, et al.
Publicado: (2020)

Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy
por: Moretti, Federico A., et al.
Publicado: (2021)

CNS Diseases and Uveitis
por: Allegri, Pia, et al.
Publicado: (2011)

Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice
por: Clarke, Don, et al.
Publicado: (2018)

Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B
por: Grover, Anita, et al.
Publicado: (2020)

Early Enzyme Replacement Therapy Improves Hearing and Immune Defects in Adenosine Deaminase Deficient-Mice
por: Xu, Xiaobai, et al.
Publicado: (2019)

EGFL7 reduces CNS inflammation in mouse
por: Larochelle, Catherine, et al.
Publicado: (2018)

Author Correction: Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy
por: Moretti, Federico A., et al.
Publicado: (2021)

Deubiquitinating enzymes (DUBs): DoUBle-edged swords in CNS autoimmunity
por: Ruan, Jing, et al.
Publicado: (2020)

CNS reservoirs for HIV: implications for eradication
por: Hellmuth, Joanna, et al.
Publicado: (2015)

EMBR-28. CNS DISTRIBUTIONAL KINETICS OF PANOBINOSTAT IN THE MOUSE
por: Zhang, Wenqiu, et al.
Publicado: (2021)

Cardiomyopathy and Response to Enzyme Replacement Therapy in a Male Mouse Model for Fabry Disease
por: Nguyen Dinh Cat, Aurelie, et al.
Publicado: (2012)

A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA
por: Sorrentino, Nicolina Cristina, et al.
Publicado: (2013)

Enzyme Replacement in Gaucher Disease
por: Beutler, Ernest
Publicado: (2004)

NFIA Haploinsufficiency Is Associated with a CNS Malformation Syndrome and Urinary Tract Defects
por: Lu, Weining, et al.
Publicado: (2007)

Multi-Agency Data Integration Project - Linking Data - Improving Lives
por: Kindermann, Bindi, et al.
Publicado: (2017)

Protein Deiminase 4 and CR3 Regulate Aspergillus fumigatus and β-Glucan-Induced Neutrophil Extracellular Trap Formation, but Hyphal Killing Is Dependent Only on CR3
por: Clark, Heather L., et al.
Publicado: (2018)

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

Dysfunction of the CNS-Heart Axis in Mouse Models of Huntington's Disease
por: Mielcarek, Michal, et al.
Publicado: (2014)

Utilizing mouse optic nerve crush to examine CNS remyelination
por: Suter, Tracey A.C.S., et al.
Publicado: (2021)

Oligodendroglia-to-neuron material transfer lights up the mouse CNS
por: Pluchino, Stefano, et al.
Publicado: (2023)

The Pharmacological Chaperone N-butyldeoxynojirimycin Enhances Enzyme Replacement Therapy in Pompe Disease Fibroblasts
por: Porto, Caterina, et al.
Publicado: (2009)

Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice
por: Padula, Agnese, et al.
Publicado: (2022)

Enzyme replacement therapy: efficacy and limitations
por: Concolino, Daniela, et al.
Publicado: (2018)

Insights into the physiological role of CNS regeneration inhibitors
por: Baldwin, Katherine T., et al.
Publicado: (2015)

CNS and CNS diseases in relation to their immune system
por: Xu, Jianhao, et al.
Publicado: (2022)

Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A
por: Esposito, Federica, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_kill37rnp0vft1dl9c76mr3g79