Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors.
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Animal models of hemophiliaComparison of factor VIII transgenes bioengineered for improved expression in gene therapy of hemophilia AStrategies to modulate immune responses: a new frontier for gene therapyAbsence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy.Advancements in gene transfer-based therapy for hemophilia A.Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model.Modifications of adenovirus hexon allow for either hepatocyte detargeting or targeting with potential evasion from Kupffer cellsProtein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.A novel cell-sheet technology that achieves durable factor VIII delivery in a mouse model of hemophilia ACorrection of hemophilia as a proof of concept for treatment of monogenic diseases by fetal spleen transplantation.Prospects for gene therapy of haemophilia.Correction of a murine model of von Willebrand disease by gene transfer.Current status and prospects for gene therapy.Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?Novel delivery methods for treatment of viral hepatitis: an update.Tolerance induction by viral in vivo gene transferTherapeutic short hairpin RNA expression in the liver: viral targets and vectors.In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCsGene therapy for treatment of inherited haematological disorders.Cellular and genetic therapies for haemophilia.Genetic engineering for haemophilia A.Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A miceIn vivo models of haemophilia - status on current knowledge of clinical phenotypes and therapeutic interventions.Gene transfer to hemophilia A mice via oral delivery of FVIII-chitosan nanoparticles.Haploidentical in utero hematopoietic cell transplantation improves phenotype and can induce tolerance for postnatal same-donor transplants in the canine leukocyte adhesion deficiency model.Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia BWFH State-of-the-Art Session on Therapeutic Gene Transfer Buenos Aires, Argentina.Historical perspective and future direction of coagulation research.Endothelial progenitor cell-based therapy for hemophilia A.Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies.Gene therapy for immune tolerance induction in hemophilia with inhibitors.Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models.A Novel Adenoviral Hybrid-vector System Carrying a Plasmid Replicon for Safe and Efficient Cell and Gene Therapeutic Applications.Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction.Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor.Advances in Overcoming Immune Responses following Hemophilia Gene Therapy.Update on gene therapy for hereditary hematological disorders.Toward gene therapy for hemophilia A with novel adenoviral vectors: successes and limitations in canine models.Factors influencing therapeutic efficacy and the host immune response to helper-dependent adenoviral gene therapy in hemophilia A mice.Gene therapy for hemophilia? Yes.The impact of antigen expression in antigen-presenting cells on humoral immune responses against the transgene product.
P2860
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P2860
Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors.
description
2002 nî lūn-bûn
@nan
2002 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2002 թվականի հոտեմբերին հրատարակված գիտական հոդված
@hy
2002年の論文
@ja
2002年論文
@yue
2002年論文
@zh-hant
2002年論文
@zh-hk
2002年論文
@zh-mo
2002年論文
@zh-tw
2002年论文
@wuu
name
Therapeutic factor VIII levels ...... h-capacity adenoviral vectors.
@ast
Therapeutic factor VIII levels ...... h-capacity adenoviral vectors.
@en
type
label
Therapeutic factor VIII levels ...... h-capacity adenoviral vectors.
@ast
Therapeutic factor VIII levels ...... h-capacity adenoviral vectors.
@en
prefLabel
Therapeutic factor VIII levels ...... h-capacity adenoviral vectors.
@ast
Therapeutic factor VIII levels ...... h-capacity adenoviral vectors.
@en
P2093
P921
P1433
P1476
Therapeutic factor VIII levels ...... h-capacity adenoviral vectors.
@en
P2093
Brian Brown
David Lillicrap
Désiré Collen
Gudrun Schiedner
Marinee K L Chuah
Marion Johnston
Nico Van Rooijen
Sabine Hertel
Stefan Kochanek
Thierry VandenDriessche
P304
P356
10.1182/BLOOD-2002-03-0823
P407
P577
2002-10-24T00:00:00Z