| P2093 | author name string | Katherine A High | |
| | Valder R Arruda | |
| | Roland W Herzog | |
| | Jane Mount | |
| | Frederik W van Ginkel | |
| | John Hathcock | |
| | D Michael Tillson | |
| | Clinton D Lothrop | |
| | Glenn P Niemeyer | |
| P2860 | cites work | Gene therapy: therapeutic gene causing lymphoma | Q28236795 |
| | Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 |
| | Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver | Q33737640 |
| | Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy | Q33770970 |
| | Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver | Q33815070 |
| | Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo | Q33844362 |
| | AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. | Q34168942 |
| | Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells | Q34574858 |
| | Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer | Q34985105 |
| | Preclinical animal models for hemophilia gene therapy: predictive value and limitations | Q35760785 |
| | Systemic protein delivery by muscle-gene transfer is limited by a local immune response | Q35847626 |
| | AAV-mediated gene transfer for treatment of hemophilia | Q36173013 |
| | Tolerance induction by viral in vivo gene transfer | Q36320026 |
| | Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. | Q36438711 |
| | Gene therapy for treatment of inherited haematological disorders | Q36446948 |
| | Cellular and genetic therapies for haemophilia | Q36473038 |
| | Emerging role of regulatory T cells in gene transfer | Q36990121 |
| | Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy. | Q40613905 |
| | AAV serotype 2 vectors preferentially integrate into active genes in mice | Q40643545 |
| | Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. | Q41722852 |
| | Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer | Q41920883 |
| | Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy. | Q42803722 |
| | Evaluation of twelve-hour preprandial and two-hour postprandial serum bile acids concentrations for diagnosis of hepatobiliary disease in dogs | Q43535685 |
| | Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors | Q43716886 |
| | Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs | Q43877932 |
| | Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates | Q44364376 |
| | Bile acid concentrations in the diagnosis of hepatobiliary disease in the dog. | Q44674982 |
| | Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. | Q44974380 |
| | Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. | Q45225518 |
| | CD8(+) T-cell responses to adeno-associated virus capsid in humans | Q45406111 |
| | Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector | Q45855552 |
| | Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs | Q45856596 |
| | Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B. | Q45857835 |
| | Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. | Q45859498 |
| | AAV vector integration sites in mouse hepatocellular carcinoma. | Q45869780 |
| | Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer | Q45874902 |
| | A deletion mutation causes hemophilia B in Lhasa Apso dogs | Q45879429 |
| | Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation | Q45879591 |
| | Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9. | Q45879597 |
| | Major role of local immune responses in antibody formation to factor IX in AAV gene transfer | Q45883516 |
| | Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy | Q45884700 |
| | Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs | Q45888388 |
| | Gene transfer as an approach to treating hemophilia | Q64377890 |
| | Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice | Q64380468 |
| | Long-term persistence of anti-factor VIII antibody-secreting cells in hemophilic mice after treatment with human factor VIII | Q74207828 |
| P433 | issue | 4 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | hemophilia | Q134003 |
| | gene therapy | Q213901 |
| | coagulation factor IX | Q425360 |
| | hemophilia B | Q2562598 |
| P304 | page(s) | 797-806 | |
| P577 | publication date | 2008-10-28 | |
| P1433 | published in | Blood | Q885070 |
| P1476 | title | Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy | |
| P478 | volume | 113 | |