Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice.
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Gene therapy for hemophiliaAbsence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy.Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.Adeno-associated virus-mediated rescue of the cognitive defects in a mouse model for Angelman syndrome.AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in miceCopackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Engineering Factor Viii for Hemophilia Gene Therapy.Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.Enhanced factor VIII heavy chain for gene therapy of hemophilia ACopackaging of multiple adeno-associated viral vectors in a single production step.Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia.MicroRNA-511 Binds to FKBP5 mRNA, Which Encodes a Chaperone Protein, and Regulates Neuronal Differentiation.Protein trans-splicing based dual-vector delivery of the coagulation factor VIII gene.Indoleamine 2,3-dioxygenase attenuates inhibitor development in gene-therapy-treated hemophilia A mice.Muscle Gene Therapy for Hemophilia.The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene.Protein trans-splicing as a means for viral vector-mediated in vivo gene therapy.Update on gene therapy for hereditary hematological disorders.Inter-chain disulfide bond improved protein trans-splicing increases plasma coagulation activity in C57BL/6 mice following portal vein FVIII gene delivery by dual vectors.
P2860
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P2860
Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice.
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2003 nî lūn-bûn
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name
Dual vectors expressing murine ...... rrection of hemophilia A mice.
@en
Dual vectors expressing murine ...... rrection of hemophilia A mice.
@nl
type
label
Dual vectors expressing murine ...... rrection of hemophilia A mice.
@en
Dual vectors expressing murine ...... rrection of hemophilia A mice.
@nl
prefLabel
Dual vectors expressing murine ...... rrection of hemophilia A mice.
@en
Dual vectors expressing murine ...... rrection of hemophilia A mice.
@nl
P2093
P921
P1433
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Dual vectors expressing murine ...... rrection of hemophilia A mice.
@en
P2093
Barry J Byrne
Cathryn Mah
Haig H Kazazian
Irene Zolotukhin
Mary Schleissing
Rita Sarkar
P304
P356
10.1089/104303403321070838
P577
2003-01-01T00:00:00Z