| scholarly article | Q13442814 |
| P356 | DOI | 10.1089/HUM.2014.153 |
| P698 | PubMed publication ID | 25675273 |
| P2093 | author name string | Mark A Kay | |
| Hansell Hall Stedman | |||
| Katherine A High | |||
| Margaret H Whitford | |||
| Timothy Nichols | |||
| Valder R Arruda | |||
| P2860 | cites work | Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 |
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| Long-term safety and efficacy of factor IX gene therapy in hemophilia B. | Q34783922 | ||
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| Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B. | Q35006608 | ||
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| Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial | Q35078437 | ||
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| Adenovirus-associated virus vector-mediated gene transfer in hemophilia B | Q35691659 | ||
| Pathways of cell infection by parvoviruses and adeno-associated viruses | Q35803571 | ||
| Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model | Q35847459 | ||
| Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer | Q35876970 | ||
| Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. | Q36007714 | ||
| Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients? | Q36230845 | ||
| Large animal models and gene therapy | Q36333340 | ||
| Glanzmann's thrombasthenia treatment: a prospective observational registry on the use of recombinant human activated factor VII and other hemostatic agents | Q36374090 | ||
| 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 | ||
| The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy. | Q36440058 | ||
| Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients | Q36812729 | ||
| Immune responses to AAV vectors: overcoming barriers to successful gene therapy | Q36983740 | ||
| Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy | Q37071092 | ||
| Review of companion animal viral diseases and immunoprophylaxis | Q37337252 | ||
| Current status of haemophilia gene therapy | Q38206852 | ||
| Adverse reproductive outcomes in urban women with adeno-associated virus-2 infections in early pregnancy | Q39819790 | ||
| Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. | Q41722852 | ||
| Toxicity and morbidity of isolated limb perfusion | Q41741351 | ||
| Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver | Q41753510 | ||
| Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer | Q41920883 | ||
| Gene therapy for hemophilia: addressing the coming challenges of affordability and accessibility | Q42541512 | ||
| Cell phones and landlines: the impact of gene therapy on the cost and availability of treatment for hemophilia | Q42933613 | ||
| Posttranslational modifications of recombinant myotube-synthesized human factor IX. | Q43513933 | ||
| Molecular genotyping of the Italian cohort of patients with hemophilia B. | Q43669780 | ||
| Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice | Q43702736 | ||
| Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector | Q43783192 | ||
| Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy | Q43801966 | ||
| Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. | Q44974380 | ||
| Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors | Q45299117 | ||
| CD8(+) T-cell responses to adeno-associated virus capsid in humans | Q45406111 | ||
| Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy | Q45409051 | ||
| Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector | Q45855552 | ||
| Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. | Q45859498 | ||
| Spectrum of F9 mutations in Chinese haemophilia B patients: identification of 20 novel mutations | Q45864776 | ||
| The special case of gene therapy pricing | Q45868383 | ||
| The human factor IX gene as germline mutagen test: samples from Mainland China have the putatively endogenous pattern of mutation | Q45868965 | ||
| AAV vector integration sites in mouse hepatocellular carcinoma. | Q45869780 | ||
| Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B. | Q45871503 | ||
| 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 | ||
| Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy | Q45884700 | ||
| Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector | Q45886806 | ||
| Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy | Q45888356 | ||
| Absence of circulating factor IX antigen in hemophilia B dogs of the UNC-Chapel Hill colony | Q74226101 | ||
| Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression | Q80141652 | ||
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | hemophilia B | Q2562598 |
| hemophilia | Q134003 | ||
| gene therapy | Q213901 | ||
| P577 | publication date | 2014-12-30 | |
| P1433 | published in | Human gene therapy. Clinical development | Q27724795 |
| P1476 | title | Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs |
| Q39546902 | Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? |
| Q64264349 | Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study |
| Q39394803 | Complexity of immune responses to AAV transgene products - Example of factor IX. |
| Q33623549 | Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models |
| Q52721830 | Gene Therapy With Regulatory T Cells: A Beneficial Alliance. |
| Q51057724 | Gene Therapy for Hemophilia. |
| Q28087557 | Gene therapy in an era of emerging treatment options for hemophilia B |
| Q37043485 | Genetic Manipulation of Brown Fat Via Oral Administration of an Engineered Recombinant Adeno-associated Viral Serotype Vector |
| Q45875499 | Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. |
| Q89777684 | Hemophilia gene therapy comes of age |
| Q45875635 | Hemophilia gene therapy comes of age. |
| Q90576641 | Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models |
| Q38529941 | Obstacles and future of gene therapy for hemophilia |
| Q37616952 | Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis. |
| Q35969194 | Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs. |
| Q36547796 | Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII |
| Q45866075 | Systemic AAV Gene Therapy Close to Clinical Trials for Several Neuromuscular Diseases |
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