Cargando…

Prevalence of Anti–Adeno-Associated Virus Serotype 8 Neutralizing Antibodies and Arylsulfatase B Cross-Reactive Immunologic Material in Mucopolysaccharidosis VI Patient Candidates for a Gene Therapy Trial

Recombinant vectors based on adeno-associated virus serotype 8 (AAV8) have been successfully used in the clinic and hold great promise for liver-directed gene therapy. Preexisting immunity against AAV8 or the development of antibodies against the therapeutic transgene product might negatively affect...

Descripción completa

Detalles Bibliográficos
Autores principales:	Ferla, Rita, Claudiani, Pamela, Savarese, Marco, Kozarsky, Karen, Parini, Rossella, Scarpa, Maurizio, Donati, Maria Alice, Sorge, Giovanni, Hopwood, John J., Parenti, Giancarlo, Fecarotta, Simona, Nigro, Vincenzo, Sivri, Hatice Serap, Van Der Ploeg, Ans, Andria, Generoso, Brunetti-Pierri, Nicola, Auricchio, Alberto
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc. 2015
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4367235/ https://www.ncbi.nlm.nih.gov/pubmed/25654180 http://dx.doi.org/10.1089/hum.2014.109

Ejemplares similares

Novel subtype of mucopolysaccharidosis caused by arylsulfatase K (ARSK) deficiency
por: Verheyen, Sarah, et al.
Publicado: (2022)

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

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

Open issues in Mucopolysaccharidosis type I-Hurler
por: Parini, Rossella, et al.
Publicado: (2017)

Hypoxia Reduces Arylsulfatase B Activity and Silencing Arylsulfatase B Replicates and Mediates the Effects of Hypoxia
por: Bhattacharyya, Sumit, et al.
Publicado: (2012)

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

Increased ocular wall thickness and decreased globe volume in children with mucopolysaccharidosis type VI
por: Yavuz, Ozlem Ozkale, et al.
Publicado: (2022)

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
por: Wraith, J. Edmond, et al.
Publicado: (2007)

Ensuring continuity of care for children with inherited metabolic diseases at the time of COVID-19: the experience of a metabolic unit in Italy
por: Brunetti-Pierri, Nicola, et al.
Publicado: (2020)

Cervical spondylolisthesis in mucopolysaccharidosis type II
por: Rossi, Alessandro, et al.
Publicado: (2022)

Accumulation of lysosulfatide in the brain of arylsulfatase A-deficient mice
por: Blomqvist, Maria, et al.
Publicado: (2011)

Distribution of Heparan Sulfate Oligosaccharides in Murine Mucopolysaccharidosis Type IIIA
por: Mason, Kerryn, et al.
Publicado: (2014)

Protein structure prediction for novel mutations in Arylsulfatase-A gene
por: Divya, M, et al.
Publicado: (2014)

Anti-diabetic activity of different oils through their effect on arylsulfatases
por: Samarji, Rima, et al.
Publicado: (2014)

Decline in arylsulfatase B leads to increased invasiveness of melanoma cells
por: Bhattacharyya, Sumit, et al.
Publicado: (2016)

Arylsulfatase A pseudodeficiency in Mexico: Enzymatic activity and haplotype analysis
por: Juárez‐Osuna, Jesús A., et al.
Publicado: (2020)

A Possible Role for Arylsulfatase G in Dermatan Sulfate Metabolism
por: Poterala-Hejmo, Aleksandra, et al.
Publicado: (2020)

Mucopolysaccharidosis type VI phenotypes-genotypes and antibody response to galsulfase
por: Brands, Marion M, et al.
Publicado: (2013)

Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI
por: Ferla, Rita, et al.
Publicado: (2017)

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

Orofacial abnormalities in mucopolysaccharidosis and mucolipidosis type II and III: A systematic review
por: de Bode, Chiel J., et al.
Publicado: (2022)

Mucopolysaccharidosis VI in cats – clarification regarding genetic testing
por: Lyons, Leslie A., et al.
Publicado: (2016)

Purification and biosynthesis of a derepressible periplasmic arylsulfatase from Chlamydomonas reinhardtii
Publicado: (1988)

Upregulation of arylsulfatase B in carotid atherosclerosis is associated with symptoms of cerebral embolization
por: Biros, Erik, et al.
Publicado: (2017)

Arylsulfatase A (ASA) in Parkinson’s Disease: From Pathogenesis to Biomarker Potential
por: Angelopoulou, Efthalia, et al.
Publicado: (2020)

Plasma arylsulfatase A levels are associated with cognitive function in Parkinson’s disease
por: Li, Mingjian, et al.
Publicado: (2022)

Mucopolysaccharidosis: Cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI
por: Brands, Marion M. M. G., et al.
Publicado: (2012)

Respiratory and sleep disorders in mucopolysaccharidosis
por: Berger, Kenneth I., et al.
Publicado: (2012)

Arylsulfatase B Improves Locomotor Function after Mouse Spinal Cord Injury
por: Yoo, Myungsik, et al.
Publicado: (2013)

Brown Algae and Basalt Meal in Maintaining the Activity of Arylsulfatase of Soil Polluted with Cadmium
por: Zaborowska, Magdalena, et al.
Publicado: (2017)

Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study
por: í Dali, Christine, et al.
Publicado: (2020)

Inhibition Properties of Arylsulfatase and β-Glucuronidase by Hydrogen Peroxide, Hypochlorite, and Peracetic Acid
por: Zhong, Shu-shu, et al.
Publicado: (2021)

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

Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease
por: Scarpa, Maurizio, et al.
Publicado: (2011)

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

Easy‐to‐use algorithm would provide faster diagnoses for mucopolysaccharidosis type I and enable patients to receive earlier treatment
por: Tylki‐Szymańska, Anna, et al.
Publicado: (2018)

Craniosynostosis affects the majority of mucopolysaccharidosis patients and can contribute to increased intracranial pressure
por: Oussoren, Esmee, et al.
Publicado: (2018)

Nonclinical comparability studies of recombinant human arylsulfatase A addressing manufacturing process changes
por: Wright, Teresa, et al.
Publicado: (2018)

Changes in plasma arylsulfatase A level as a compensatory biomarker of early Parkinson’s disease
por: Yoo, Han Soo, et al.
Publicado: (2020)

Three novel variants in the arylsulfatase A (ARSA) gene in patients with metachromatic leukodystrophy (MLD)
por: Hettiarachchi, D., et al.
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_cplglkj3j3f1sshr148fmesqbk