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8
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9
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Hum Gene Ther
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18
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Clin Exp Immunol
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Polarized expression of bone morphogenetic protein-4 in the human aorta-gonad-mesonephros region.
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Blood
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Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2Rd2/Rd2 model of retinal degeneration.
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Gene Ther
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Cutting edge: the Wiskott-Aldrich syndrome protein is required for efficient phagocytosis of apoptotic cells.
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J Immunol
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Hum Gene Ther
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Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells.
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Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX.
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Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS.
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Intrinsic dendritic cell abnormalities in Wiskott-Aldrich syndrome.
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Adeno-associated virus gene transfer to mouse retina.
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Hum Gene Ther
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Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina.
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Translational mini-review series on immunodeficiency: molecular defects in common variable immunodeficiency.
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Clin Exp Immunol
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Progress and prospects: gene therapy for inherited immunodeficiencies.
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Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors.
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Normal development of human fetal hematopoiesis between eight and seventeen weeks' gestation.
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Am J Obstet Gynecol
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Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression.
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Detailed characterization of the human aorta-gonad-mesonephros region reveals morphological polarity resembling a hematopoietic stromal layer.
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0.98
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Adeno-associated and herpes simplex viruses as vectors for gene transfer to the corneal endothelium.
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High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H.
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Cell-specific and efficient expression in mouse and human B cells by a novel hybrid immunoglobulin promoter in a lentiviral vector.
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Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer.
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Improved antitumour immunity in murine neuroblastoma using a combination of IL-2 and IL-12.
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A defined window for efficient gene marking of severe combined immunodeficient-repopulating cells using a gibbon ape leukemia virus-pseudotyped retroviral vector.
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Pure populations of transduced primary human cells can be produced using GFP expressing herpes virus vectors and flow cytometry.
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Critical variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency.
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High efficiency gene transfer to human hematopoietic SCID-repopulating cells under serum-free conditions.
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Optimization of recombinant adeno-associated virus production using an herpes simplex virus amplicon system.
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