Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy.
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MicroRNAs in platelet production and activationMicroRNA-regulated viral vectors for gene therapyGene therapy for primary immunodeficienciesMicroRNA-restricted transgene expression in the retinaGenetic engineering of hematopoiesis for targeted IFN-α delivery inhibits breast cancer progressionLysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular ToxicityDevelopment of a New Tool for 3D Modeling for Regenerative MedicineHematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice.Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal diseaseCombined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.Gene-microRNA network module analysis for ovarian cancer.Gene therapy for PIDs: progress, pitfalls and prospectsTherapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophyEctopic miR-125a Expression Induces Long-Term Repopulating Stem Cell Capacity in Mouse and Human Hematopoietic ProgenitorsGammaretroviral vectors: biology, technology and applicationJoint analysis of miRNA and mRNA expression data.Exploiting microRNA regulation for genetic engineering.High-throughput assessment of microRNA activity and function using microRNA sensor and decoy libraries.Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.Experimental therapies in the murine model of globoid cell leukodystrophy.Gene therapy for lysosomal storage disorders: a good start.Dynamic Activity of miR-125b and miR-93 during Murine Neural Stem Cell Differentiation In Vitro and in the Subventricular Zone Neurogenic Niche.Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Gene therapy for the nervous system: challenges and new strategies.Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophyNeural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy.Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.The myelin mutants as models to study myelin repair in the leukodystrophies.Functional Studies of microRNAs in Neural Stem Cells: Problems and PerspectivesProgenitor cell-based treatment of the pediatric myelin disordersDesign of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophyAttenuation of miR-126 activity expands HSC in vivo without exhaustion.miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells.Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing.Transient Expression of an LEDGF/p75 Chimera Retargets Lentivector Integration and Functionally Rescues in a Model for X-CGD.MicroRNA-223 dose levels fine tune proliferation and differentiation in human cord blood progenitors and acute myeloid leukemia.Molecular Analysis of Neutrophil Differentiation from Human Induced Pluripotent Stem Cells Delineates the Kinetics of Key Regulators of Hematopoiesis.Human miR223 promoter as a novel myelo-specific promoter for chronic granulomatous disease gene therapyMissense mutation in mouse GALC mimics human gene defect and offers new insights into Krabbe diseaseHematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects.
P2860
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P2860
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy.
description
2010 nî lūn-bûn
@nan
2010年の論文
@ja
2010年学术文章
@wuu
2010年学术文章
@zh
2010年学术文章
@zh-cn
2010年学术文章
@zh-hans
2010年学术文章
@zh-my
2010年学术文章
@zh-sg
2010年學術文章
@yue
2010年學術文章
@zh-hant
name
Identification of hematopoieti ...... f globoid cell leukodystrophy.
@en
Identification of hematopoieti ...... f globoid cell leukodystrophy.
@nl
type
label
Identification of hematopoieti ...... f globoid cell leukodystrophy.
@en
Identification of hematopoieti ...... f globoid cell leukodystrophy.
@nl
prefLabel
Identification of hematopoieti ...... f globoid cell leukodystrophy.
@en
Identification of hematopoieti ...... f globoid cell leukodystrophy.
@nl
P2093
P2860
P50
P1476
Identification of hematopoieti ...... f globoid cell leukodystrophy.
@en
P2093
Aldo Orlacchio
Alessandra Biffi
Eric Lechman
Giulia Schira
Hidefumi Hiramatsu
Ilaria Visigalli
Silvia Ungari
P2860
P304
P356
10.1126/SCITRANSLMED.3001522
P407
P50
P577
2010-11-01T00:00:00Z