Cargando…

Toward a gene therapy for neurological and somatic MPSIIIA

Mucopolysaccharidosis Type IIIA (MPSIIIA) represents an unmet medical need. MPSIIIA shares with many other lysosomal storage disorders (LSD) the characteristic of being a severe neurodegenerative disease accompanied by mild somatic involvement. Thus, the main target organ for the development of new...

Descripción completa

Detalles Bibliográficos
Autores principales:	Haurigot, Virginia, Bosch, Fatima
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Landes Bioscience 2013
Materias:	Addendum
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3927492/ https://www.ncbi.nlm.nih.gov/pubmed/25003015 http://dx.doi.org/10.4161/rdis.27209

Ejemplares similares

A step closer toward therapies for p63-related disorders
por: Zhou, Huiqing, et al.
Publicado: (2013)

Problems and solutions for the analysis of somatic CAG repeat expansion and their relationship to Huntington's disease toxicity
por: Budworth, Helen, et al.
Publicado: (2016)

Addendum: da Silva, J.L.F.; et al. The Lisbon Supramolecular Green Story: Mechanochemistry towards New Forms of Pharmaceuticals. Molecules 2020, 25, 2705
por: da Silva, João Luís Ferreira, et al.
Publicado: (2021)

Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis
por: Braun, Christian Joerg, et al.
Publicado: (2014)

Toward a porcine in vivo model to analyze the pathogenesis of TLR5-dependent enteropathies
por: Pieper, Robert, et al.
Publicado: (2020)

Microbial signatures in post-infectious irritable bowel syndrome – toward patient stratification for improved diagnostics and treatment
por: Jalanka, Jonna, et al.
Publicado: (2015)

Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC
por: Marcó, Sara, et al.
Publicado: (2016)

Addendum: Immune-related adverse events and atypical radiological response with checkpoint inhibitor immunotherapy in an elderly patient with high PD-L1 expressing lung adenocarcinoma
por: Teixidor, Eduard, et al.
Publicado: (2023)

Addendum: Cechová, M. et al. Towards Better Understanding of Pea Seed Dormancy Using Laser Desorption/Ionization Mass Spectrometry. Int. J. Mol. Sci. 2017, 18, 2196
por: Cechová, Monika, et al.
Publicado: (2017)

Addendum: Shin, W.; et al. The Knowledge and Attitude of Patients Diagnosed with Epithelial Ovarian Cancer towards Genetic Testing. Int. J. Environ. Res. Public Health 2021, 18, 2312
por: Shin, Wonkyo, et al.
Publicado: (2021)

Breaking restricted taxonomic functionality by dual resistance genes
por: Narusaka, Mari, et al.
Publicado: (2013)

Addendum: ROS1-GOPC/FIG: a novel gene fusion in hepatic angiosarcoma
por: Marks, Eric I., et al.
Publicado: (2023)

Addendum: A case report of androgen receptor inhibitor therapy in recurrent high-grade serous ovarian cancer
por: Limaye, Sewanti, et al.
Publicado: (2022)

Addendum: Immunotherapy in Xeroderma Pigmentosum: a case of advanced cutaneous squamous cell carcinoma treated with cemiplimab and a literature review
por: Rubatto, Marco, et al.
Publicado: (2022)

Muscle-specific microRNAs as biomarkers of Duchenne Muscular Dystrophy progression and response to therapies
por: Giordani, L, et al.
Publicado: (2014)

Addendum: Resolution of radiation necrosis with bevacizumab following radiation therapy for primary CNS lymphoma
por: Vaios, Eugene J., et al.
Publicado: (2022)

Gene regulation networks generate diverse pigmentation patterns in plants
por: Albert, Nick W, et al.
Publicado: (2014)

Addendum: Precise tuning of gene expression levels in mammalian cells
por: Michaels, Yale S., et al.
Publicado: (2019)

Editorial Expression of Concern: Transgenic Aedes aegypti Mosquitoes Transfer Genes into a Natural Population
por: Evans, Benjamin R., et al.
Publicado: (2020)

Beyond multiple mechanisms and a unique drug: Defective autophagy as pivotal player in cerebral cavernous malformation pathogenesis and implications for targeted therapies
por: Marchi, Saverio, et al.
Publicado: (2016)

Brain connectivity changes occurring following cognitive behavioural therapy for psychosis predict long-term recovery
por: Mason, L, et al.
Publicado: (2017)

Pigmentation-based insertional mutagenesis is a simple and potent screening approach for identifying neurocristopathy-associated genes in mice
por: Pilon, Nicolas
Publicado: (2016)

Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease
por: Guo, Shuling, et al.
Publicado: (2014)

Editorial Expression of Concern: birinapant sensitizes platinum-resistant carcinomas with high levels of cIAP to carboplatin therapy
por: La, V., et al.
Publicado: (2018)

Addendum to ‘A comparison of electronically-delivered and face to face cognitive behavioural therapies in depressive disorders: A systematic review and meta-analysis’ [EClinicalMedicine 24 (2020) 100442]
por: Luo, Candice, et al.
Publicado: (2020)

Motor neuron degeneration in spinal and Bulbar Muscular Atrophy is a skeletal muscle-driven process: Relevance to therapy development and implications for related motor neuron diseases
por: Cortes, Constanza J, et al.
Publicado: (2014)

Periodic mRNA synthesis and degradation co‐operate during cell cycle gene expression
por: Eser, Philipp, et al.
Publicado: (2014)

What do we learn from the murine Jacob/Nsmf gene knockout for human disease?
por: Spilker, Christina, et al.
Publicado: (2016)

Direct activation of EXPANSIN14 by LBD18 in the gene regulatory network of lateral root formation in Arabidopsis
por: Kim, Jungmook, et al.
Publicado: (2013)

Addendum: Sustained response of three pediatric BRAF(V600E) mutated high-grade gliomas to combined BRAF and MEK inhibitor therapy
por: Toll, Stephanie A., et al.
Publicado: (2023)

Silence is golden: gene silencing of V. cholerae during intestinal colonization delivers new aspects to the acid tolerance response
por: Cakar, Fatih, et al.
Publicado: (2018)

Addendum to the Acknowledgements: Video-Delivered Family Therapy for Home Visited Young Mothers With Perinatal Depressive Symptoms: Quasi-Experimental Implementation-Effectiveness Hybrid Trial
por: Cluxton-Keller, Fallon, et al.
Publicado: (2019)

Addendum: Liquid biopsy and multi-analyte testing guided treatment of HER2 positive periampullary adenocarcinoma with durable complete response after trastuzumab based therapy
por: Nagarkar, Rajnish, et al.
Publicado: (2022)

Addendum: Iwaszko et al., Influence of NKG2D Genetic Variants on Response to Anti-TNF Agents in Patients with Rheumatoid Arthritis. Genes 2018, 9, 64
por: Iwaszko, Milena, et al.
Publicado: (2018)

Addendum: Amplification, Sequencing and Cloning of Iranian Native Bacillus subtilis Alpha-amylase Gene in Saccharomyces cerevisiae [published in Jundishapur J Microbiol. 2013;6(8):e7371]
por: Afzal-Javan, Fahimeh, et al.
Publicado: (2014)

Shiga toxin 2a and Enteroaggregative Escherichia coli – a deadly combination
por: Boisen, Nadia, et al.
Publicado: (2015)

A commentary on the utility of a new L-DOPA-responsive dystonia mouse model
por: Rose, Samuel J., et al.
Publicado: (2015)

Clozapine in a patient with treatment-resistant schizophrenia and hypertrophic cardiomyopathy: a case report — ADDENDUM
por: Sanchez, Asiel Yair Adan, et al.
Publicado: (2019)

Editorial Expression of Concern: IRE1a constitutes a negative feedback loop with BMP2 and acts as a novel mediator in modulating osteogenic differentiation
por: Guo, F. J., et al.
Publicado: (2018)

Staufen1s role as a splicing factor and a disease modifier in Myotonic Dystrophy Type I
por: Bondy-Chorney, Emma, et al.
Publicado: (2016)

Cannot write session to /tmp/vufind_sessions/sess_0tfvid47vequfv1n2d7p1avdde