Results 31 to 40 of about 29,065 (252)

Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX

Cell Reports, 2018
Summary: Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient ...
Suvasini Ramaswamy, Nina Tonnu, Tushar Menon, Benjamin M. Lewis, Kevin T. Green, Derek Wampler, Paul E. Monahan, Inder M. Verma   +7 more
doaj   +1 more source

Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A.

, 2012
nefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production.
Tonn, Torsten, Milanov, Peter, Schüttrumpf, Jörg, Seifried, Erhard, Patricia Quade-Lyssy, Quade-Lyssy, Patricia, Jörg Schüttrumpf, Roth, Stefanie D., Simpson, Jeremy C., Christopher Ungerer, Erhard Seifried, Abriss, Daniela, Pepperkok, Rainer, Rainer Pepperkok, Stefanie D Roth, Jeremy C Simpson, Torsten Tonn, Ungerer, Christopher, Peter Milanov, Daniela Abriss   +19 more
core   +1 more source

Haemophilia B: an illustrative review of current challenges and opportunities

Research and Practice in Thrombosis and Haemostasis
Background: Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX, which presents unique challenges in clinical management.
Cedric Hermans, Jan Astermark, Sonata Šaulytė Trakymienė, Víctor Jiménez-Yuste   +3 more
doaj   +1 more source

Dental Management under General anesthesia of Children with Hemophilia A and Hemophilia B [PDF]

, 2018
Hemophilia is an X-linked herediatry disorder. Hemophilia A is a defiency of factor VIII and hemophilia B (Christmas disease) is a deficiency of factor IX. The most widely used classification is based on plasma procoagulant levels, with people 〈1% factor
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core   +1 more source

The molecular basis of hemophilia B

, 2022
Hemophilia B (HB) is a genetically determined bleeding disorder characterized by deficiency of the coagulation factor IX (FIX). The severity of bleeding phenotype is associated with FIX plasma level.
Odnoczko, Edyta; Department of Hemostasis and Metabolic Disorders, Institute of Hematology and Transfusion Medicine, Warsaw, Malarczyk, Daria; Department of Hemostasis and Metabolic Disorders, Institute of Hematology and Transfusion Medicine, Warsaw, Adamiec, Agata; Department of Hemostasis and Metabolic Disorders, Institute of Hematology and Transfusion Medicine, Warsaw   +2 more
core   +1 more source

Clinical profile of hemophilia B patients from Karnataka

, 2022
Background: The most prevalent severe inherited hemorrhagic condition is hemophilia, which means “love of blood.” Hemophilia A and B are caused by a lack or malfunction of the factor VIII and factor IX proteins.
Pramod B Gai, Smita Hegde, Sanjeev Kolagi, Suresh Hanagvadi, Sujayendra Kulkarni, Rajat Hegde, Kusal K Das, Suyamindra S Kulkarni, Rudragouda S Bulagouda   +8 more
core   +1 more source

Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy

Molecular Therapy: Methods & Clinical Development, 2019
Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively.
Benjamin J. Samelson-Jones, Valder R. Arruda   +1 more
doaj   +1 more source

Exploring the Burden on Patients Living With and Receiving Treatment for Immune Thrombocytopenia (ITP): Patient and Physician Perceptions From the ITP World Impact Survey (I‐WISh) 2.0

American Journal of Hematology, EarlyView.
ABSTRACT Limited data exist on how patients and physicians perceive immune thrombocytopenia (ITP) symptoms and treatment‐related burden. I‐WISh (ITP World Impact Survey) 2.0 surveyed 1018 patients and 431 physicians in 15 countries to characterize the impact of ITP and its treatments on patients.
Nichola Cooper, James Bussel, Waleed Ghanima, Drew Provan, Yoshiaki Tomiyama, Ming Hou, Donald M. Arnold, Cristina Santoro, Francesco Zaja, Barbara Lovrencic, Mervyn Morgan, Michal Winograd, Jennifer DiRaimo, Danielle Boyle, Olivera Rajkovic‐Hooley, Meritxell Vendranas, Susan Frade, Caroline Kruse   +17 more
wiley   +1 more source

Adenovirus-based gene therapy approaches for hemophilia B [PDF]

, 2012
Gene therapy can be used to treat devastating inherited diseases, especially diseases and patients that are not suitable for a conventional cure. The blood clotting disorder hemophilia is one of the most extensively studied monogenetic diseases in gene ...
Zhang, Wenli
core  

The Molecular Basis of FIX Deficiency in Hemophilia B

, 2022
Coagulation factor IX (FIX) is a vitamin K dependent protein and its deficiency causes hemophilia B, an X-linked recessive bleeding disorder. More than 1000 mutations in the F9 gene have been identified in hemophilia B patients.
Weikai Li, Guomin Shen, Meng Gao, Qing Cao   +3 more
core   +1 more source