Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens.
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Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B miceIn utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical applicationHemophilia A: an ideal disease to correct in uteroAnimal models of hemophiliaHemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectorsGene therapy for hemophiliaClinical and molecular characterization of a re-established line of sheep exhibiting hemophilia A.Comparison of factor VIII transgenes bioengineered for improved expression in gene therapy of hemophilia ADirected engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A.Advancements in gene transfer-based therapy for hemophilia A.In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infectionIn vitro and In vivo Model Systems for Hemophilia A Gene Therapy.Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy.Expanding the ortholog approach for hemophilia treatment complicated by factor VIII inhibitorsEnhanced biosynthesis of coagulation factor VIII through diminished engagement of the unfolded protein response.Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.Phenotypic correction of hemophilia A in sheep by postnatal intraperitoneal transplantation of FVIII-expressing MSC.Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene DeliveryGeneration of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia AEffects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A.Combination of double negative T cells and anti-thymocyte serum reverses type 1 diabetes in NOD miceSyngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunityComparison of human coagulation factor VIII expression directed by cytomegalovirus and mammary gland-specific promoters in HC11 cells and transgenic miceInduction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy ProductsCorrection of murine hemophilia A following nonmyeloablative transplantation of hematopoietic stem cells engineered to encode an enhanced human factor VIII variant using a safety-augmented retroviral vector.Anti-CD3 antibodies modulate anti-factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy.Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells.Delivery of nucleic acid therapeutics by genetically engineered hematopoietic stem cells.Immunomodulation for inhibitors in hemophilia A: the important role of Treg cellsObstacles and future of gene therapy for hemophilia.Gene therapy for immune tolerance induction in hemophilia with inhibitors.State of the art: gene therapy of haemophilia.Indoleamine 2,3-dioxygenase attenuates inhibitor development in gene-therapy-treated hemophilia A mice.In Vivo Hematopoietic Stem Cell Transduction.Advances in Overcoming Immune Responses following Hemophilia Gene Therapy.Contribution of ADAMTS13 to the better cell engraftment efficacy in mouse model of bone marrow transplantationTilt balance towards regulation: evolving new strategy for treatment of hemophilia inhibitors.Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A.Immune tolerance induced by platelet-targeted factor VIII gene therapy in hemophilia A mice is CD4 T cell mediated.Platelet-directed gene therapy overcomes inhibitory antibodies to factor VIII.
P2860
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P2860
Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens.
description
2007 nî lūn-bûn
@nan
2007年の論文
@ja
2007年論文
@yue
2007年論文
@zh-hant
2007年論文
@zh-hk
2007年論文
@zh-mo
2007年論文
@zh-tw
2007年论文
@wuu
2007年论文
@zh
2007年论文
@zh-cn
name
Hematopoietic stem-cell gene t ...... blative conditioning regimens.
@ast
Hematopoietic stem-cell gene t ...... blative conditioning regimens.
@en
type
label
Hematopoietic stem-cell gene t ...... blative conditioning regimens.
@ast
Hematopoietic stem-cell gene t ...... blative conditioning regimens.
@en
prefLabel
Hematopoietic stem-cell gene t ...... blative conditioning regimens.
@ast
Hematopoietic stem-cell gene t ...... blative conditioning regimens.
@en
P2093
P2860
P921
P1433
P1476
Hematopoietic stem-cell gene t ...... blative conditioning regimens.
@en
P2093
Bagirath Gangadharan
Christopher B Doering
H Trent Spencer
Kuang-Yueh Chiang
Lucienne M Ide
P2860
P304
P356
10.1182/BLOOD-2007-04-082602
P407
P577
2007-06-14T00:00:00Z