| scholarly article | Q13442814 |
| P50 | author | James Wilson | Q16947166 |
| P2093 | author name string | L Wang | |
| Y Li | |||
| D Wu | |||
| P Bell | |||
| J-P Louboutin | |||
| J A Greig | |||
| P2860 | cites work | Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 |
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. | Q31934499 | ||
| Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle | Q33713019 | ||
| Hepatic gene transfer as a means of tolerance induction to transgene products | Q33775537 | ||
| AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. | Q34168942 | ||
| Clades of Adeno-associated viruses are widely disseminated in human tissues. | Q34193756 | ||
| Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer | Q34985105 | ||
| Immunity to adenovirus and adeno-associated viral vectors: implications for gene therapy | Q35131227 | ||
| Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates | Q35628868 | ||
| Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model | Q35847459 | ||
| Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver | Q35848563 | ||
| Recombinant adeno-associated virus vectors induce functionally impaired transgene product-specific CD8+ T cells in mice | Q36144935 | ||
| Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus | Q36159178 | ||
| New recombinant serotypes of AAV vectors. | Q36172991 | ||
| 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 | ||
| Sustained correction of bleeding disorder in hemophilia B mice by gene therapy | Q36454880 | ||
| A factor IX-deficient mouse model for hemophilia B gene therapy | Q36602472 | ||
| Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors | Q36734619 | ||
| Enhancing transduction of the liver by adeno-associated viral vectors | Q37042394 | ||
| AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells | Q37348251 | ||
| Role of regulatory T cells in tolerance to coagulation factors | Q37561470 | ||
| Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer | Q39302902 | ||
| Induction of immune tolerance to FIX following muscular AAV gene transfer is AAV-dose/FIX-level dependent | Q39506153 | ||
| Gene therapy vectors based on adeno-associated virus type 1. | Q39550492 | ||
| Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. | Q41722852 | ||
| Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver | Q41753510 | ||
| Vaccines based on novel adeno-associated virus vectors elicit aberrant CD8+ T-cell responses in mice | Q42121728 | ||
| Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets | Q42613058 | ||
| Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors | Q43716886 | ||
| Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy | Q43801966 | ||
| Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy | Q44405190 | ||
| Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs | Q44540545 | ||
| Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors | Q45299117 | ||
| Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid | Q45414481 | ||
| Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia a dogs and mice | Q45417773 | ||
| Phenotype correction of hemophilia A mice with adeno-associated virus vectors carrying the B domain-deleted canine factor VIII gene. | Q45421368 | ||
| Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector | Q45855552 | ||
| In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor | Q45855716 | ||
| Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. | Q45859498 | ||
| Persistent expression of canine factor IX in hemophilia B canines | Q45862802 | ||
| Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter. | Q45866541 | ||
| Efficient induction of immune tolerance to coagulation factor IX following direct intramuscular gene transfer | Q45866661 | ||
| Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. | Q45869438 | ||
| Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX. | Q45869966 | ||
| Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors | Q45873434 | ||
| Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. | Q45875077 | ||
| Gene transfer into skeletal muscle using novel AAV serotypes. | Q45879206 | ||
| 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 | ||
| Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors | Q45879602 | ||
| Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. | Q45882349 | ||
| 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 | ||
| Human factor IX corrects the bleeding diathesis of mice with hemophilia B. | Q45885256 | ||
| Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy | Q45888356 | ||
| P433 | issue | 10 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | hemophilia | Q134003 |
| gene therapy | Q213901 | ||
| hemophilia B | Q2562598 | ||
| P304 | page(s) | 2009-2019 | |
| P577 | publication date | 2011-10-01 | |
| P1433 | published in | Journal of Thrombosis and Haemostasis | Q6296004 |
| P1476 | title | Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors | |
| P478 | volume | 9 |
| Q37690491 | AAV8 induces tolerance in murine muscle as a result of poor APC transduction, T cell exhaustion, and minimal MHCI upregulation on target cells. |
| Q35184217 | Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero delivery of adeno-associated virus vector serotype 2/9 |
| Q35418068 | Intramuscular injection of AAV8 in mice and macaques is associated with substantial hepatic targeting and transgene expression |
| Q37051733 | Multilineage transduction of resident lung cells in vivo by AAV2/8 for α1-antitrypsin gene therapy |
| Q37066342 | Multiple recombinant adeno-associated viral vector serotypes display persistent in vivo gene expression in vector-transduced rat stifle joints |
| Q37690441 | Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice |
| Q37621530 | Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodies |
| Q38168940 | Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera®). |
| Q90390816 | Superior human hepatocyte transduction with adeno-associated virus vector serotype 7 |
| Q40318313 | Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs |
| Q35876224 | The AAV vector toolkit: poised at the clinical crossroads |
| Q33902499 | The potential of adeno-associated viral vectors for gene delivery to muscle tissue |
| Q36538166 | Treatment of hypophosphatasia by muscle-directed expression of bone-targeted alkaline phosphatase via self-complementary AAV8 vector |
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