Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
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Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B miceConcise review: stem cell-derived erythrocytes as upcoming players in blood transfusionThe potential of stem cells as an in vitro source of red blood cells for transfusionAnimal models of hemophiliaGene therapy for hemophiliaEfficient hepatocyte engraftment and long-term transgene expression after reversible portal embolization in nonhuman primates.Comparison of factor VIII transgenes bioengineered for improved expression in gene therapy of hemophilia ACell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidosesGenomic and functional assays demonstrate reduced gammaretroviral vector genotoxicity associated with use of the cHS4 chromatin insulator.Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice.Development of Gene Transfer for Induction of Antigen-specific Tolerance.Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector.Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.Lentiviral vectors incorporating a human elongation factor 1alpha promoter for the treatment of canine leukocyte adhesion deficiency.Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment.Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras.Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells.Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I.Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome.Bioengineering of differentiated hepatocytes with human factor IX-expressing plasmids in vitro.Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells.Recent advances in lentiviral vector development and applications.Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice.Gene therapy for haemophilia: a long and winding road.De novo formed satellite DNA-based mammalian artificial chromosomes and their possible applications.New and improved AAVenues: current status of hemophilia B gene therapy.A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectorsSickle cells produce functional immune modulators and cytotoxics.Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in beta-thalassemic mice.Production of functional coagulation factor VIII from iPSCs using a lentiviral vector.Platelet-directed gene therapy overcomes inhibitory antibodies to factor VIII.Intra-articular injection of mesenchymal stem cells expressing coagulation factor ameliorates hemophilic arthropathy in factor VIII-deficient mice.High efficacy and safety of low-dose CD19-directed CAR-T cell therapy in 51 refractory or relapsed B acute lymphoblastic leukemia patients.Factor VIII delivered by haematopoietic stem cell-derived B cells corrects the phenotype of haemophilia A mice.
P2860
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P2860
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
description
2006 nî lūn-bûn
@nan
2006年の論文
@ja
2006年学术文章
@wuu
2006年学术文章
@zh
2006年学术文章
@zh-cn
2006年学术文章
@zh-hans
2006年学术文章
@zh-my
2006年学术文章
@zh-sg
2006年學術文章
@yue
2006年學術文章
@zh-hant
name
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
@en
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
@nl
type
label
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
@en
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
@nl
prefLabel
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
@en
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
@nl
P2093
P2860
P356
P1433
P1476
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
@en
P2093
Alex H Chang
Matthias T Stephan
Michel Sadelain
P2860
P2888
P304
P356
10.1038/NBT1227
P577
2006-07-16T00:00:00Z
P5875
P6179
1025618915