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1. Full Text Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
by Nathwani, Amit C and Reiss, Ulreke M and Tuddenham, Edward G.D and Rosales, Cecilia and Chowdary, Pratima and McIntosh, Jenny and Della Peruta, Marco and Lheriteau, Elsa and Patel, Nishal and Raj, Deepak and Riddell, Anne and Pie, Jun and Rangarajan, Savita and Bevan, David and Recht, Michael and Shen, Yu-Min and Halka, Kathleen G and Basner-Tschakarjan, Etiena and Mingozzi, Federico and High, Katherine A and Allay, James and Kay, Mark A and Ng, Catherine Y.C and Zhou, Junfang and Cancio, Maria and Morton, Christopher L and Gray, John T and Srivastava, Deokumar and Nienhuis, Arthur W and Davidoff, Andrew M
The New England journal of medicine, ISSN 0028-4793, 11/2014, Volume 371, Issue 21, pp. 1994 - 2004
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Hematologic and hematopoietic diseases | Biological and medical sciences | General aspects | Medical sciences | Platelet diseases and coagulopathies | Hemophilia B - therapy | Dependovirus - genetics | Gene Expression | Follow-Up Studies | Genetic Vectors - administration & dosage | Humans | Middle Aged | Hemophilia B - blood | Factor IX - genetics | Male | Alanine Transaminase - blood | Hemophilia B - genetics | Young Adult | Adult | Factor IX - metabolism | Infusions, Intravenous | Transgenes | Genetic Therapy - adverse effects | Physiological aspects | Hemophilia B | Research | Gene expression | Safety education | Analysis | Alanine | Intravenous administration | Toxicity | Genes | Body weight | Genomes | Patients | Clinical outcomes | Coagulation factors | Hemophilia | Alanine transaminase | Gene therapy | Prednisolone | Index Medicus | Abridged Index Medicus
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2. Full Text Severe hemophilia with mild bleeding phenotype: molecular characterization and global coagulation profile
by SANTAGOSTINO, E and MANCUSO, M. E and TRIPODI, A and CHANTARANGKUL, V and CLERICI, M and GARAGIOLA, I and MANNUCCI, P. M
Journal of thrombosis and haemostasis, ISSN 1538-7933, 04/2010, Volume 8, Issue 4, pp. 737 - 743
phenotype | hemophilia A | endogenous thrombin potential | hemophilia B | genotype | Genotype | Hemophilia B | Phenotype | Endogenous thrombin potential | Hemophilia A | Peripheral Vascular Disease | Life Sciences & Biomedicine | Hematology | Cardiovascular System & Cardiology | Science & Technology | Predictive Value of Tests | Hemophilia B - diagnosis | Humans | Middle Aged | Case-Control Studies | Hemorrhage - etiology | Adult | Factor IX - metabolism | Hemophilia A - diagnosis | Odds Ratio | Severity of Illness Index | Genetic Predisposition to Disease | Hemorrhage - genetics | Risk Assessment | Gene Frequency | Risk Factors | Hemophilia B - blood | Factor VIII - genetics | Factor IX - genetics | Logistic Models | Hemorrhage - blood | Hemophilia B - genetics | Hemophilia A - blood | Hemophilia A - complications | Hemophilia B - complications | Blood Coagulation Tests | Blood Coagulation - genetics | Italy | Hemophilia A - genetics | Factor VIII - metabolism | Thrombin - metabolism | Genetic aspects | Blood coagulation factors | Analysis | Hemophilia | Index Medicus
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3. Full Text Definitions in hemophilia: communication from the SSC of the ISTH
by Blanchette, V. S and Key, N. S and Ljung, L. R and Manco‐Johnson, M. J and Berg, H. M and Srivastava, A and Subcomm Factor VIII Factor IX Rare and Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis and the Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders
Journal of thrombosis and haemostasis, ISSN 1538-7933, 11/2014, Volume 12, Issue 11, pp. 1935 - 1939
Peripheral Vascular Disease | Life Sciences & Biomedicine | Hematology | Cardiovascular System & Cardiology | Science & Technology | Hemophilia B - therapy | Severity of Illness Index | Hemarthrosis - diagnosis | Hemophilia B - classification | Predictive Value of Tests | Hemophilia B - diagnosis | Humans | Hemophilia B - blood | Terminology as Topic | Treatment Outcome | Hemophilia A - therapy | Hemophilia A - blood | Disease Progression | Hemarthrosis - blood | Hemarthrosis - classification | Coagulants - therapeutic use | Hemophilia A - classification | Hemarthrosis - therapy | Hemophilia A - diagnosis | Hemophilia | Index Medicus | Kardiologi | Clinical Medicine | Cardiac and Cardiovascular Systems | Medical and Health Sciences | Klinisk medicin | Medicin och hälsovetenskap
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4. Full Text Hemophilia B: molecular pathogenesis and mutation analysis
by Goodeve, A. C
Journal of thrombosis and haemostasis, ISSN 1538-7933, 07/2015, Volume 13, Issue 7, pp. 1184 - 1195
genetic carrier detection | factor IX | prenatal diagnosis | DNA Mutational Analysis | hemophilia B | Hemophilia B | DNA mutational analysis | Factor IX | Genetic carrier detection | Prenatal diagnosis | Severity of Illness Index | Predictive Value of Tests | Genetic Predisposition to Disease | Hemophilia B - diagnosis | Prognosis | Humans | Prenatal Diagnosis | Risk Factors | Hemophilia B - blood | Factor IX - genetics | Hemostasis - genetics | Male | Hemophilia B - genetics | Phenotype | Animals | Sex Factors | Factor IX - metabolism | Female | Heterozygote | Mutation | Algorithms | Pregnant women | Analysis | Hemophilia | Genetic aspects | Nucleotide sequencing | Prothrombin complex concentrate | DNA sequencing | Index Medicus | Review
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5. Full Text Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
by George, Lindsey A and Sullivan, Lisa M and Sullivan, Spencer K and Giermasz, Adam and Rasko, John E.J and Samelson-Jones, Benjamin J and Ducore, Jonathan and Cuker, Adam and Majumdar, Suvankar and Teitel, Jerome and McGuinn, Catherine E and Ragni, Margaret V and Luk, Alvin Y and Hui, Daniel and Wright, J. Fraser and Chen, Yifeng and Liu, Yun and Wachtel, Katie and Winters, Angela and Tiefenbacher, Stefan and Arruda, Valder R and van der Loo, Johannes C.M and Zelenaia, Olga and Takefman, Daniel and Carr, Marcus E and Couto, Linda B and Anguela, Xavier M and High, Katherine A
The New England journal of medicine, ISSN 0028-4793, 12/2017, Volume 377, Issue 23, pp. 2215 - 2227
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Hemophilia B - therapy | Genetic Vectors - administration & dosage | Factor IX - therapeutic use | Humans | Middle Aged | Dependovirus - immunology | Factor IX - genetics | Male | Hemorrhage - prevention & control | Hemophilia B - genetics | Young Adult | Adolescent | Adult | Factor IX - metabolism | Transgenes | Hemophilia B - metabolism | Genetic Therapy - methods | Usage | Genetic aspects | Research | Gene therapy | Genetic variation | Hemophilia | Coagulation factors | Liver | Body weight | Prophylaxis | Prednisone | Genomes | Drug dosages | Bleeding | Clotting | Index Medicus | Abridged Index Medicus
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6. Full Text Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B
by Miesbach, Wolfgang and Meijer, Karina and Coppens, Michiel and Kampmann, Peter and Klamroth, Robert and Schutgens, Roger and Tangelder, Marco and Castaman, Giancarlo and Schwäble, Joachim and Bonig, Halvard and Seifried, Erhard and Cattaneo, Federica and Meyer, Christian and Leebeek, Frank W. G
Blood, ISSN 0006-4971, 2018, Volume 131, Issue 9, pp. 1022 - 1031
Life Sciences & Biomedicine | Hematology | Science & Technology | Hemophilia B - therapy | Genetic Therapy | Prednisolone - administration & dosage | Humans | Hemophilia B - blood | Factor IX - genetics | Male | Hemophilia B - genetics | Adult | Female | Genetic Vectors | Factor IX - biosynthesis | Parvovirinae | Index Medicus | Abridged Index Medicus | Thrombosis and Hemostasis
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7. Full Text Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
by Pasi, K. John and Rangarajan, Savita and Georgiev, Pencho and Mant, Tim and Creagh, Michael D and Lissitchkov, Toshko and Bevan, David and Austin, Steve and Hay, Charles R and Hegemann, Inga and Kazmi, Rashid and Chowdary, Pratima and Gercheva-Kyuchukova, Liana and Mamonov, Vasily and Timofeeva, Margarita and Soh, Chang-Heok and Garg, Pushkal and Vaishnaw, Akshay and Akinc, Akin and Sørensen, Benny and Ragni, Margaret V
The New England journal of medicine, ISSN 0028-4793, 08/2017, Volume 377, Issue 9, pp. 819 - 828
Hemophilia B - therapy | Single-Blind Method | Humans | Middle Aged | Hemophilia B - blood | Male | Healthy Volunteers | Hemophilia A - therapy | Hemophilia A - blood | Dose-Response Relationship, Drug | Young Adult | Thrombin - biosynthesis | Antithrombin III - antagonists & inhibitors | Injections, Subcutaneous | Antithrombins - blood | Adult | RNAi Therapeutics | Care and treatment | Usage | Antithrombin III | Hemophilia | Hemophilia B | Research | Gene therapy | Plasma | Intravenous administration | Hematology | RNA-mediated interference | Liver | Body weight | Thrombin | Injection | Antithrombin | Clotting | Plasma levels | Alloantibodies | Thromboembolism | Drug dosages | Index Medicus | Abridged Index Medicus
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8. Full Text Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
by Buchlis, George and Podsakoff, Gregory M and Radu, Antonetta and Hawk, Sarah M and Flake, Alan W and Mingozzi, Federico and High, Katherine A
Blood, ISSN 0006-4971, 03/2012, Volume 119, Issue 13, pp. 3038 - 3041
Life Sciences & Biomedicine | Hematology | Science & Technology | Hematologic and hematopoietic diseases | Biological and medical sciences | Medical sciences | Platelet diseases and coagulopathies | Hemophilia B - therapy | Severity of Illness Index | Dependovirus - genetics | Gene Transfer Techniques | Gene Expression | Genetic Vectors - administration & dosage | Humans | Factor IX - genetics | Male | Muscle, Skeletal - metabolism | Hemophilia B - genetics | Genetic Vectors - genetics | Dependovirus - physiology | Organ Specificity - genetics | Hemophilia B - pathology | Time Factors | Adult | Factor IX - metabolism | Muscle, Skeletal - pathology | Hemophilia B - metabolism | Genetic Therapy - methods | Index Medicus | Abridged Index Medicus | Thrombosis and Hemostasis | Gene Therapy | Brief Report
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9. Full Text Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
by Niemeyer, Glenn P and Herzog, Roland W and Mount, Jane and Arruda, Valder R and Tillson, D. Michael and Hathcock, John and Van Ginkel, Frederik W and High, Katherine A and Lothrop Jr, Clinton D
Blood, ISSN 0006-4971, 01/2009, Volume 113, Issue 4, pp. 797 - 806
Life Sciences & Biomedicine | Hematology | Science & Technology | Hematologic and hematopoietic diseases | Biological and medical sciences | Medical sciences | Platelet diseases and coagulopathies | Hemophilia B - therapy | Dependovirus - genetics | Liver - metabolism | RNA, Messenger - genetics | Gene Expression Regulation | Factor IX - genetics | Tomography, X-Ray Computed | Hemophilia B - genetics | Genetic Vectors - genetics | Animals | Hemophilia B - pathology | Time Factors | Immune Tolerance - immunology | Dogs | Factor IX - metabolism | DNA, Viral - genetics | Hemophilia B - metabolism | Genetic Therapy - adverse effects | Index Medicus | Abridged Index Medicus | Gene Therapy
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10. Full Text The past and future of haemophilia: diagnosis, treatments, and its complications
by Peyvandi, Flora, Prof and Garagiola, Isabella, PhD and Young, Guy, MD
The Lancet (British edition), ISSN 0140-6736, 2016, Volume 388, Issue 10040, pp. 187 - 197
Internal Medicine | Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Hemophilia B - therapy | Factor VIII - immunology | Genetic Therapy | Hemophilia B - diagnosis | Hemophilia B - immunology | Factor IX - immunology | Factor IX - therapeutic use | Hemorrhage - therapy | Hemophilia A - immunology | Humans | Hemorrhage - prevention & control | Hemophilia A - therapy | Coagulants - therapeutic use | Antibodies, Neutralizing - immunology | Early Medical Intervention | Coagulants - immunology | Hemophilia A - diagnosis | Factor VIII - therapeutic use | Complications and side effects | Medical colleges | Hemophilia | Prothrombin | Thrombin | Diagnosis | Plants | Genes | Medical treatment | Clinical trials | Mutation | Disease management | Medical diagnosis | Index Medicus | Abridged Index Medicus
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