Advances toward gene therapy for hemophilia at the millennium.
about
Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model.Delivery of factor VIII gene into skeletal muscle cells using lentiviral vector.In vivo efficacy of platelet-delivered, high specific activity factor VIII variantsGene therapy for liver diseases: recent strategies for treatment of viral hepatitis and liver malignanciesIn vitro and In vivo Model Systems for Hemophilia A Gene Therapy.Inhibitor development in correlation to factor VIII genotypes.Parvovirus-mediated gene transfer for the haemophilias.Hemophilia: treatment options in the twenty-first century.Intensive exposure to factor VIII is a risk factor for inhibitor development in mild hemophilia A.The future of recombinant coagulation factors.Apoptotic effects of platelet factor VIII on megakaryopoiesis: implications for a modified human FVIII for platelet-based gene therapy.Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer.Incorporation of DOPE into Lipoplexes formed from a Ferrocenyl Lipid leads to Inverse Hexagonal Nanostructures that allow Redox-Based Control of Transfection in High Serum.AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in 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 immunityComplete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette.Phenotypic correction of murine hemophilia A using an iPS cell-based therapyGene transfer to hemophilia A mice via oral delivery of FVIII-chitosan nanoparticles.Enhanced factor VIII heavy chain for gene therapy of hemophilia AMannose-dependent endoplasmic reticulum (ER)-Golgi intermediate compartment-53-mediated ER to Golgi trafficking of coagulation factors V and VIII.Protein trans-splicing based dual-vector delivery of the coagulation factor VIII gene.Long-term correction of hemophilia A mice following lentiviral mediated delivery of an optimized canine factor VIII gene.The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene.Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.A chamber of hope for hemophilia.Factor IX gene sequencing by a simple and sensitive 15-hour procedure for haemophilia B diagnosis: identification of two novel mutations.Allergenic vaccines administration and inhibitor development in haemophilia.Pharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B.Engraftment of genetically modified human amniotic fluid-derived progenitor cells to produce coagulation factor IX after in utero transplantation in mice.
P2860
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P2860
Advances toward gene therapy for hemophilia at the millennium.
description
1999 nî lūn-bûn
@nan
1999 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած
@hyw
1999 թվականի սեպտեմբերին հրատարակված գիտական հոդված
@hy
1999年の論文
@ja
1999年論文
@yue
1999年論文
@zh-hant
1999年論文
@zh-hk
1999年論文
@zh-mo
1999年論文
@zh-tw
1999年论文
@wuu
name
Advances toward gene therapy for hemophilia at the millennium.
@ast
Advances toward gene therapy for hemophilia at the millennium.
@en
type
label
Advances toward gene therapy for hemophilia at the millennium.
@ast
Advances toward gene therapy for hemophilia at the millennium.
@en
prefLabel
Advances toward gene therapy for hemophilia at the millennium.
@ast
Advances toward gene therapy for hemophilia at the millennium.
@en
P921
P1433
P1476
Advances toward gene therapy for hemophilia at the millennium.
@en
P2093
Kaufman RJ
P304
P356
10.1089/10430349950017095
P577
1999-09-01T00:00:00Z