Results 11 to 20 of about 14,739,736 (367)

Encapsulation of factor IX–engineered mesenchymal stem cells in fibrinogen–alginate microcapsules enhances their viability and transgene secretion

Journal of Tissue Engineering, 2012
Cell microencapsulation holds significant promise as a strategy for cellular therapies; however, inadequate survival and functionality of the enclosed cells limit its application in hemophilia treatment.
Bahareh Sayyar, Megan Dodd, Jianping Wen, Shirley Ma, Leah Marquez-Curtis, Anna Janowska-Wieczorek, Gonzalo Hortelano   +6 more
doaj   +2 more sources

Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua. [PDF]

J Thromb Haemost, 2021
Limited information exists regarding the factor IX (FIX) coagulant activity (FIX:C) measured by different assays following FIX‐Padua gene therapy.
Robinson MM, George LA, Carr ME, Samelson-Jones BJ, Arruda VR, Murphy JE, Rybin D, Rupon J, High KA, Tiefenbacher S.   +9 more
europepmc   +2 more sources

Factor IX

Reactions Weekly, 2014
F. Giannelli
semanticscholar   +7 more sources

Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B

Current Medical Research and Opinion, 2022
Objective Congenital hemophilia B is a rare bleeding disorder caused by defects in the gene encoding factor IX (FIX) leading to coagulation deficiency. Recurrent bleeds may cause chronic pain, disability, and reduced quality of life.
Jinesh Shah, Hongseok Kim, K. Sivamurthy, P. Monahan, Michael Fries   +4 more
semanticscholar   +1 more source

Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B [PDF]

Blood, 2011
Claude Négrier, Karin Knobe, Andreas Tiede, Paul Giangrande, Judi Møss   +4 more
openalex   +2 more sources

A Case of Heel Reconstruction with a Reverse Sural Artery Flap in a Hemophilia B Patient

Archives of Plastic Surgery, 2012
Hemophilia B is a rare blood coagulation disorder. Complications such as bleeding and hematoma can cause necrosis of flaps, wound disruption, and the disturbance of wound healing.
Byung Kwon Lee, Jeong Su Shim
doaj   +1 more source

Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs

Molecular Therapy: Methods & Clinical Development, 2019
Gene therapy for severe hemophilia B is advancing and offers sustained disease amelioration with a single treatment. We have reported the efficacy and safety of AMT-060, an investigational gene therapy comprising an adeno-associated virus serotype 5 ...
Elisabeth A. Spronck, Ying Poi Liu, Jacek Lubelski, Erich Ehlert, Sander Gielen, Paula Montenegro-Miranda, Martin de Haan, Bart Nijmeijer, Valerie Ferreira, Harald Petry, Sander J. van Deventer   +10 more
doaj   +1 more source

X-Ray Determination of the Variable Rate of Mass Accretion onto TW Hydrae [PDF]

, 2012
Diagnostics of electron temperature (T_e), electron density (n_e), and hydrogen column density (N_H) from the Chandra High Energy Transmission Grating spectrum of He-like Ne IX in TW Hydrae (TW Hya), in conjunction with a classical accretion model, allow
Brickhouse, N. S., Cranmer, S. R., Dupree, A. K., Günther, H. M., Luna, G. J. M., Wolk, S. J.   +5 more
core   +2 more sources

Design of a Prospective Study on Pharmacokinetic-Guided Dosing of Prophylactic Factor Replacement in Hemophilia A and B (OPTI-CLOT TARGET Study)

TH Open, 2022
In resource-rich countries, almost all severe hemophilia patients receive prophylactic replacement therapy with factor concentrates to prevent spontaneous bleeding in joints and muscles to decrease the development of arthropathy and risk of long-term ...
Tine M.H.J. Goedhart, Laura H. Bukkems, Michiel Coppens, Karin J. Fijnvandraat, Saskia E.M. Schols, Roger E.G. Schutgens, Jeroen Eikenboom, Floor C.J.I. Heubel-Moenen, Paula F. Ypma, L. Nieuwenhuizen, K. Meijer, Frank W. G. Leebeek, Ron A.A. Mathôt, Marjon H. Cnossen   +13 more
doaj   +1 more source

Inhibitors in Patients with Congenital Bleeding Disorders Other Than Hemophilia [PDF]

, 2017
The most worrying complication of replacement therapy for severe hemophilia A and B is currently the occurrence of inhibitory alloantibodies against infused factor VIII and factor IX, respectively.
Franchini, Massimo, Liumbruno, Giancarlo Maria, Marano, Giuseppe, Mengoli, Carlo, Piccinini, Vanessa, Pupella, Simonetta, Vaglio, Stefania   +6 more
core   +1 more source