المؤلفون: Childers KC; Chemistry Department, Western Washington University, Bellingham, Washington, USA., Cowper B; Medicines and Healthcare products Regulatory Agency, South Mimms Laboratories, Potters Bar, Hertfordshire, UK., Vaughan JD; Chemistry Department, Western Washington University, Bellingham, Washington, USA., McGill JR; Chemistry Department, Western Washington University, Bellingham, Washington, USA., Davulcu O; Pacific Northwest Center for Cryo-EM, Oregon Health & Science University, Portland, Oregon, USA; Pacific Northwest National Laboratory, Environmental Molecular Sciences Laboratory, Richland, Washington, USA., Lollar P; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia, USA; Department of Pediatrics, Emory University, Atlanta, Georgia, USA., Doering CB; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia, USA; Department of Pediatrics, Emory University, Atlanta, Georgia, USA; Expression Therapeutics, Inc, Tucker, Georgia, USA., Coxon CH; Medicines and Healthcare products Regulatory Agency, South Mimms Laboratories, Potters Bar, Hertfordshire, UK., Spiegel PC Jr; Chemistry Department, Western Washington University, Bellingham, Washington, USA. Electronic address: paul.spiegel@wwu.edu.

المصدر: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Sep; Vol. 22 (9), pp. 2449-2459. Date of Electronic Publication: 2024 Jun 05.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

مواضيع طبية MeSH: Factor VIII*/immunology , Factor VIII*/chemistry , Factor VIII*/metabolism , Hemophilia A*/immunology , Hemophilia A*/drug therapy , Epitopes*/chemistry , Protein Binding*, Humans ; Animals ; Protein Domains ; von Willebrand Factor/metabolism ; von Willebrand Factor/chemistry ; Cryoelectron Microscopy ; Models, Molecular ; Structure-Activity Relationship ; Mice ; Binding Sites ; Protein Conformation ; Blood Coagulation

المؤلفون: Tonetto E; Department of Life Sciences and Biotechnology and Laboratorio per le Tecnologie delle Terapie Avanzate (LTTA), University of Ferrara, Ferrara, Italy., Cucci A; Department of Health Sciences, School of Medicine, University of Piemonte Orientale, Novara, Italy., Follenzi A; Department of Health Sciences, School of Medicine, University of Piemonte Orientale, Novara, Italy., Bernardi F; Department of Life Sciences and Biotechnology and Laboratorio per le Tecnologie delle Terapie Avanzate (LTTA), University of Ferrara, Ferrara, Italy., Pinotti M; Department of Life Sciences and Biotechnology and Laboratorio per le Tecnologie delle Terapie Avanzate (LTTA), University of Ferrara, Ferrara, Italy. Electronic address: pnm@unife.it., Balestra D; Department of Life Sciences and Biotechnology and Laboratorio per le Tecnologie delle Terapie Avanzate (LTTA), University of Ferrara, Ferrara, Italy.

المصدر: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Aug; Vol. 22 (8), pp. 2171-2183. Date of Electronic Publication: 2024 May 07.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

مواضيع طبية MeSH: Factor VIII*/genetics , Factor VIII*/metabolism , Hemophilia A*/genetics , Hemophilia A*/therapy , Hemophilia A*/blood , Gene Editing*/methods , Genetic Therapy*, Humans ; HEK293 Cells ; Endothelial Cells/metabolism ; Point Mutation ; Mutation ; CRISPR-Cas Systems

المؤلفون: Stoutenburg E; Department of Medicine, Larner College of Medicine at the University of Vermont, Burlington, Vermont, USA., Sherman S; Department of Medicine, Tulane University School of Medicine, New Orleans, Louisiana, USA., Bravo M; Department of Biochemistry, Larner College of Medicine at the University of Vermont, Burlington, Vermont, USA., Howard V; Department of Epidemiology, University of Alabama at Birmingham, Birmingham, Alabama, USA., Mukaz DK; Department of Medicine, Larner College of Medicine at the University of Vermont, Burlington, Vermont, USA., Cushman M; Department of Medicine, Larner College of Medicine at the University of Vermont, Burlington, Vermont, USA.; Department of Pathology and Laboratory Science, Larner College of Medicine at the University of Vermont, Burlington, Vermont, USA., Zakai NA; Department of Medicine, Larner College of Medicine at the University of Vermont, Burlington, Vermont, USA.; Department of Pathology and Laboratory Science, Larner College of Medicine at the University of Vermont, Burlington, Vermont, USA., Judd S; Department of Biostatistics, University of Alabama at Birmingham, Birmingham, Alabama, USA., Plante TB; Department of Medicine, Larner College of Medicine at the University of Vermont, Burlington, Vermont, USA.

المصدر: American journal of hypertension [Am J Hypertens] 2024 Jul 15; Vol. 37 (8), pp. 580-587.

نوع المنشور: Journal Article; Multicenter Study; Comparative Study

بيانات الدورية: Publisher: Oxford University Press Country of Publication: United States NLM ID: 8803676 Publication Model: Print Cited Medium: Internet ISSN: 1941-7225 (Electronic) Linking ISSN: 08957061 NLM ISO Abbreviation: Am J Hypertens Subsets: MEDLINE

مواضيع طبية MeSH: Factor VIII*/analysis , Factor VIII*/metabolism , Health Status Disparities* , Hypertension*/ethnology , Hypertension*/epidemiology , Hypertension*/physiopathology, Aged ; Female ; Humans ; Male ; Middle Aged ; Biomarkers/blood ; Black or African American ; Blood Pressure ; Incidence ; Prospective Studies ; Risk Assessment ; Risk Factors ; Stroke/ethnology ; Stroke/epidemiology ; Stroke/blood ; United States/epidemiology ; White

المؤلفون: Kim MS; School of Pharmacy, University of Waterloo, Kitchener, Ontario, Canada., Hajducek DM; School of Pharmacy, University of Waterloo, Kitchener, Ontario, Canada., Gilbert JC; Band Therapeutics, Lexington, Massachusetts, USA., Iorio A; Department of Medicine, McMaster University, Hamilton, Ontario, Canada., Jilma B; Department of Clinical Pharmacology, Medical University of Vienna, Vienna, Austria., Edginton AN; School of Pharmacy, University of Waterloo, Kitchener, Ontario, Canada. aedginto@uwaterloo.ca.

المصدر: The AAPS journal [AAPS J] 2024 Jul 12; Vol. 26 (4), pp. 81. Date of Electronic Publication: 2024 Jul 12.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: American Association of Pharmaceutical Scientists Country of Publication: United States NLM ID: 101223209 Publication Model: Electronic Cited Medium: Internet ISSN: 1550-7416 (Electronic) Linking ISSN: 15507416 NLM ISO Abbreviation: AAPS J Subsets: MEDLINE

مواضيع طبية MeSH: Factor VIII*/pharmacokinetics , Factor VIII*/administration & dosage , Hemophilia A*/drug therapy , Hemophilia A*/blood , von Willebrand Factor*/pharmacokinetics , von Willebrand Factor*/administration & dosage, Humans ; Models, Biological ; Adult ; Male ; Young Adult ; Kinetics ; Polyethylene Glycols/pharmacokinetics ; Polyethylene Glycols/administration & dosage ; Adolescent

المؤلفون: Long BR; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA. Electronic address: brian.long@bmrn.com., Robinson TM; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Day JRS; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Yu H; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Lau K; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Imtiaz U; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Patton KS; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., de Hart G; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Henshaw J; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Agarwal S; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Vettermann C; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Zoog SJ; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Gupta S; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.

المصدر: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Jul 03; Vol. 32 (7), pp. 2052-2063. Date of Electronic Publication: 2024 May 24.

نوع المنشور: Journal Article; Clinical Trial, Phase III

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/therapy , Hemophilia A*/immunology , Hemophilia A*/genetics , Dependovirus*/genetics , Dependovirus*/immunology , Genetic Therapy*/methods , Genetic Vectors*/genetics , Genetic Vectors*/administration & dosage , Factor VIII*/genetics , Factor VIII*/immunology, Humans ; Male ; Adult ; Treatment Outcome ; Transgenes ; Young Adult ; Antibodies, Viral/immunology ; Antibodies, Viral/blood ; Middle Aged

SCR Organism: Adeno-associated virus-5

المؤلفون: Hermans C; Division of Haematology, Haemostasis and Thrombosis Unit, Saint-Luc University Hospital, Université catholique de Louvain (UCLouvain), Brussels, Belgium; World Federation of Hemophilia, Montreal, Quebec, Canada. Electronic address: cedric.hermans@uclouvain.be., Pierce GF; World Federation of Hemophilia, Montreal, Quebec, Canada.

المصدر: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Jul; Vol. 22 (7), pp. 1844-1846. Date of Electronic Publication: 2024 Apr 27.

نوع المنشور: Journal Article; Review

بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/blood , Hemophilia A*/drug therapy , Factor VIII*/pharmacokinetics , Factor VIII*/administration & dosage , Factor VIII*/therapeutic use, Humans ; von Willebrand Factor/metabolism ; Half-Life ; Coagulants/pharmacokinetics ; Coagulants/therapeutic use ; Coagulants/administration & dosage ; Immunoglobulin Fc Fragments ; Treatment Outcome ; Hemostasis/drug effects ; Infusions, Intravenous ; Blood Coagulation/drug effects ; Animals ; Recombinant Fusion Proteins/therapeutic use ; Recombinant Fusion Proteins/pharmacokinetics ; Recombinant Fusion Proteins/administration & dosage

المؤلفون: Zheng Q; Department of Hematology, Fujian Institute of Hematology, Fujian Provincial Key Laboratory of Hematology, Fujian Medical University Union Hospital, Fuzhou, Fujian, China., Lin K; Department of Hematology, Fujian Institute of Hematology, Fujian Provincial Key Laboratory of Hematology, Fujian Medical University Union Hospital, Fuzhou, Fujian, China., Zhang N; Department of Hematology, Fujian Institute of Hematology, Fujian Provincial Key Laboratory of Hematology, Fujian Medical University Union Hospital, Fuzhou, Fujian, China., Shi Q; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA; Blood Research Institute, Versiti, Milwaukee, WI, USA., Wu Y; Department of Hematology, Fujian Institute of Hematology, Fujian Provincial Key Laboratory of Hematology, Fujian Medical University Union Hospital, Fuzhou, Fujian, China. Electronic address: wuyong9195@126.com., Chen Y; Department of Hematology, Fujian Institute of Hematology, Fujian Provincial Key Laboratory of Hematology, Fujian Medical University Union Hospital, Fuzhou, Fujian, China; Medical Technology and Engineering College of Fujian Medical University, Fuzhou, Fujian, China; Key Laboratory of Clinical Laboratory Technology for Precision Medicine (Fujian Medical University), Fujian Province University, Fuzhou, China. Electronic address: chenyingyu@yahoo.com.

المصدر: International immunopharmacology [Int Immunopharmacol] 2024 Sep 30; Vol. 139, pp. 112735. Date of Electronic Publication: 2024 Jul 26.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Elsevier Science Country of Publication: Netherlands NLM ID: 100965259 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-1705 (Electronic) Linking ISSN: 15675769 NLM ISO Abbreviation: Int Immunopharmacol Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/drug therapy , Hemophilia A*/immunology , Factor VIII*/immunology , Antibodies, Monoclonal*/therapeutic use , Antibodies, Monoclonal*/pharmacology , Antibodies, Monoclonal*/immunology , Chemokine CXCL13*/immunology , Chemokine CXCL13*/metabolism, Animals ; Mice ; Humans ; Antigens, CD20/immunology ; Disease Models, Animal ; Mice, Inbred C57BL ; Male

المؤلفون: Calvo-Villas JM; Hematology Department, University Hospital Miguel Servet, Zaragoza, Spain., Núñez-Vázquez R; Hemophilia Unit, University Hospital Virgen del Rocío, Sevilla, Spain., Benítez-Hidalgo O; Hematology Department, University Hospital Vall d'Hebron, Barcelona, Spain., García-Díaz C; Hematology Department, University Hospital of Burgos, Spain., Galmés B; Hematology Department, University Hospital Son Espases, Palma de Mallorca, Spain., Carvalho M; Congenital Coagulopathies Reference Centre, São João University Hospital Centre, Porto, Portugal., Serrano-Torres P; Commonwealth Serum Laboratories (CSL) Behring, Barcelona, Spain., Aznar-Salatti J; Commonwealth Serum Laboratories (CSL) Behring, Barcelona, Spain., Álvarez-Román MT; Department of Hematology, La Paz University Hospital, Madrid, Spain.

المصدر: Medicine [Medicine (Baltimore)] 2024 Sep 06; Vol. 103 (36), pp. e39255.

نوع المنشور: Journal Article; Multicenter Study; Observational Study

بيانات الدورية: Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 2985248R Publication Model: Print Cited Medium: Internet ISSN: 1536-5964 (Electronic) Linking ISSN: 00257974 NLM ISO Abbreviation: Medicine (Baltimore) Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/drug therapy , Factor VIII*/therapeutic use , Factor VIII*/administration & dosage, Humans ; Retrospective Studies ; Adult ; Male ; Spain ; Young Adult ; Portugal ; Adolescent ; Middle Aged ; Hemorrhage ; Treatment Outcome ; Child ; Drug Substitution ; Female

المؤلفون: Kolyadko VN; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA. Electronic address: kolyadkov@chop.edu.

المصدر: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Sep; Vol. 22 (9), pp. 2416-2418.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

مواضيع طبية MeSH: Factor VIII*/immunology , Factor VIII*/antagonists & inhibitors , Hemophilia A*/drug therapy , Hemophilia A*/blood , Hemophilia A*/immunology, Humans ; Blood Coagulation/drug effects ; Animals ; Blood Coagulation Factor Inhibitors/blood ; Blood Coagulation Factor Inhibitors/immunology

المؤلفون: Maarouf M; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, Georgia, United States., Patel SR; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, Georgia, United States., Baldwin WH; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, Georgia, United States., Zerra PE; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, Georgia, United States.; Department of Laboratory Medicine and Pathology, Center for Transfusion Medicine and Cellular Therapies, Emory University School of Medicine, Atlanta, Georgia, United States., Cox C; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, Georgia, United States., Parker ET; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, Georgia, United States., Stowell SR; Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, United States., Meeks SL; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, Georgia, United States.

المصدر: Thrombosis and haemostasis [Thromb Haemost] 2024 Sep; Vol. 124 (9), pp. 893-896. Date of Electronic Publication: 2024 Jul 16.

نوع المنشور: Letter; Journal Article

بيانات الدورية: Publisher: Thieme Country of Publication: Germany NLM ID: 7608063 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2567-689X (Electronic) Linking ISSN: 03406245 NLM ISO Abbreviation: Thromb Haemost Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/immunology , Hemophilia A*/blood , Factor VIII*/immunology , Factor VIII*/metabolism , B-Lymphocytes*/immunology , Disease Models, Animal*, Animals ; Mice ; Mice, Knockout ; Mice, Inbred C57BL