Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy.
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Gene therapy for hemophiliaImproved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice.Strategies to modulate immune responses: a new frontier for gene therapyHepatic gene transfer as a means of tolerance induction to transgene productsIn vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infectionTargeting FVIII expression to endothelial cells regenerates a releasable pool of FVIII and restores hemostasis in a mouse model of hemophilia A.Platelets as delivery systems for disease treatmentsEradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy.Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.Design of the INHIBIT trial: preventing inhibitors by avoiding 'danger', prolonging half-life and promoting tolerance.Correction of hemophilia as a proof of concept for treatment of monogenic diseases by fetal spleen transplantation.Perinatal gene transfer to the liver.Intravascular recovery of VWF and FVIII following intraperitoneal injection and differences from intravenous and subcutaneous injection in mice.Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity.Tolerance induction by viral in vivo gene transferGene therapy for treatment of inherited haematological disorders.Genetic engineering for haemophilia A.Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment.Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunityLong-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy.Animal models of hemophilia and related bleeding disorders.Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice.Mesenchymal stem cells contribute to endogenous FVIII:c production.Obstacles and future of gene therapy for hemophilia.Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies.Characterization of four novel molecular changes in the promoter region of the factor VIII gene.Indoleamine 2,3-dioxygenase attenuates inhibitor development in gene-therapy-treated hemophilia A mice.Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.Gene Therapy for Hemophilia: Progress to Date.Gene therapy for hemophilia: what does the future hold?
P2860
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P2860
Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy.
description
2005 nî lūn-bûn
@nan
2005 թուականի Ապրիլին հրատարակուած գիտական յօդուած
@hyw
2005 թվականի ապրիլին հրատարակված գիտական հոդված
@hy
2005年の論文
@ja
2005年論文
@yue
2005年論文
@zh-hant
2005年論文
@zh-hk
2005年論文
@zh-mo
2005年論文
@zh-tw
2005年论文
@wuu
name
Absence of a desmopressin resp ...... irected neonatal gene therapy.
@ast
Absence of a desmopressin resp ...... irected neonatal gene therapy.
@en
type
label
Absence of a desmopressin resp ...... irected neonatal gene therapy.
@ast
Absence of a desmopressin resp ...... irected neonatal gene therapy.
@en
prefLabel
Absence of a desmopressin resp ...... irected neonatal gene therapy.
@ast
Absence of a desmopressin resp ...... irected neonatal gene therapy.
@en
P2093
P2860
P921
P356
P1476
Absence of a desmopressin resp ...... irected neonatal gene therapy.
@en
P2093
Dwight A Bellinger
Katherine P Ponder
Lingfei Xu
Rita Sarkar
Stephanie McCorquodale
Timothy C Nichols
P2860
P304
P356
10.1073/PNAS.0409249102
P407
P577
2005-04-18T00:00:00Z