Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease
about
Checkpoints to the Brain: Directing Myeloid Cell Migration to the Central Nervous SystemRecommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.Quantitative assessment of barriers to the clinical development and adoption of cellular therapies: A pilot studyGene therapy for lysosomal storage disorders: a good start.Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I.Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier.Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Gene therapy for neurologic manifestations of mucopolysaccharidoses.Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome.Clinical applications involving CNS gene transfer.Methods for gene transfer to the central nervous system.Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene deliveryEnzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres.Gene therapy for the neurological manifestations in lysosomal storage disorders.Potential mechanisms for cell-based gene therapy to treat HIV/AIDS.Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB.Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice.How close are we to therapies for Sanfilippo disease?Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.HIV-1 infection of microglial cells in a reconstituted humanized mouse model and identification of compounds that selectively reverse HIV latency.A novel conditional Sgsh knockout mouse model recapitulates phenotypic and neuropathic deficits of Sanfilippo syndrome.High content screening of patient-derived cell lines highlights the potential of non-standard chemotherapeutic agents for the treatment of glioblastoma.A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms.Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy
P2860
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P2860
Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease
description
2013 nî lūn-bûn
@nan
2013 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2013 թվականի հունիսին հրատարակված գիտական հոդված
@hy
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
name
Myeloid/Microglial driven auto ...... euronopathic lysosomal disease
@ast
Myeloid/Microglial driven auto ...... euronopathic lysosomal disease
@en
type
label
Myeloid/Microglial driven auto ...... euronopathic lysosomal disease
@ast
Myeloid/Microglial driven auto ...... euronopathic lysosomal disease
@en
prefLabel
Myeloid/Microglial driven auto ...... euronopathic lysosomal disease
@ast
Myeloid/Microglial driven auto ...... euronopathic lysosomal disease
@en
P2093
P2860
P356
P1433
P1476
Myeloid/Microglial driven auto ...... euronopathic lysosomal disease
@en
P2093
Alexander Langford-Smith
Ana Sergijenko
Brian W Bigger
Catherine L R Merry
Claire E Pickford
Fiona L Wilkinson
Gabriel Nowinski
J Edmond Wraith
John McDermott
P2860
P304
P356
10.1038/MT.2013.141
P577
2013-06-07T00:00:00Z