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Gene therapy for haemophiliaGene therapy for haemophiliaIn 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 hemophiliaGenome-editing technologies for gene correction of hemophiliaLiver-targeted gene therapy: Approaches and challengesMulticenter trial of desirudin for the prophylaxis of thrombosis: an alternative to heparin-based anticoagulation (DESIR-ABLE).The potential of adeno-associated viral vectors for gene delivery to muscle tissue.In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infectionTransient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy.Inhibitors - cellular aspects and novel approaches for tolerance.Correction of Diabetic Hyperglycemia and Amelioration of Metabolic Anomalies by Minicircle DNA Mediated Glucose-Dependent Hepatic Insulin Production.AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transductionThe efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene DeliveryMinicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level.Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.Preliminary study on non-viral transfection of F9 (factor IX) gene by nucleofection in human adipose-derived mesenchymal stem cellsMinimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype.Bioengineering of AAV2 capsid at specific serine, threonine, or lysine residues improves its transduction efficiency in vitro and in vivo.Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX.Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells.Biological therapies for von Willebrand disease.Management of joint bleeding in hemophilia.Will gene therapy trump factor treatment in hemophilia?Biological therapies for inherited diseases: social and bioethical considerations. Hemophilia as an example.RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice.Muscle Gene Therapy for Hemophilia.A simplified purification protocol for recombinant adeno-associated virus vectors.AAV Delivery of Endothelin-1 shRNA Attenuates Cold-Induced Hypertension.Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome.AAV vector biology in primates: finding the missing link?Immune tolerance induced by platelet-targeted factor VIII gene therapy in hemophilia A mice is CD4 T cell mediated.Intra-articular injection of mesenchymal stem cells expressing coagulation factor ameliorates hemophilic arthropathy in factor VIII-deficient mice.A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model.
P2860
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description
article científic
@ca
article scientifique
@fr
articolo scientifico
@it
artigo científico
@pt
bilimsel makale
@tr
scientific article published on July 2011
@en
vedecký článok
@sk
vetenskaplig artikel
@sv
videnskabelig artikel
@da
vědecký článek
@cs
name
Gene therapy for haemophilia: a long and winding road.
@en
Gene therapy for haemophilia: a long and winding road.
@nl
type
label
Gene therapy for haemophilia: a long and winding road.
@en
Gene therapy for haemophilia: a long and winding road.
@nl
prefLabel
Gene therapy for haemophilia: a long and winding road.
@en
Gene therapy for haemophilia: a long and winding road.
@nl
P2860
P1476
Gene therapy for haemophilia: a long and winding road
@en
P2093
P2860
P356
10.1111/J.1538-7836.2011.04369.X
P478
P577
2011-07-01T00:00:00Z