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Items: 1 to 20 of 163


Incorporation of the factor IX Padua mutation into FIX-Triple improves clotting activity in vitro and in vivo.

Kao CY, Yang SJ, Tao MH, Jeng YM, Yu IS, Lin SW.

Thromb Haemost. 2013 Aug;110(2):244-56. doi: 10.1160/TH13-02-0154.


FIX-Triple, a gain-of-function factor IX variant, improves haemostasis in mouse models without increased risk of thrombosis.

Kao CY, Lin CN, Yu IS, Tao MH, Wu HL, Shi GY, Yang YL, Kao JT, Lin SW.

Thromb Haemost. 2010 Aug;104(2):355-65. doi: 10.1160/TH09-11-0792.


Expression and characterization of a novel human recombinant factor IX molecule with enhanced in vitro and in vivo clotting activity.

Perot E, Enjolras N, Le Quellec S, Indalecio A, Girard J, Negrier C, Dargaud Y.

Thromb Res. 2015 May;135(5):1017-24. doi: 10.1016/j.thromres.2015.02.034.


Generation of a novel factor IX with augmented clotting activities in vitro and in vivo.

Lin CN, Kao CY, Miao CH, Hamaguchi N, Wu HL, Shi GY, Liu YL, High KA, Lin SW.

J Thromb Haemost. 2010 Aug;8(8):1773-83. doi: 10.1111/j.1538-7836.2010.03913.x.


AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.

Crudele JM, Finn JD, Siner JI, Martin NB, Niemeyer GP, Zhou S, Mingozzi F, Lothrop CD Jr, Arruda VR.

Blood. 2015 Mar 5;125(10):1553-61. doi: 10.1182/blood-2014-07-588194.


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.

Monahan PE, Sun J, Gui T, Hu G, Hannah WB, Wichlan DG, Wu Z, Grieger JC, Li C, Suwanmanee T, Stafford DW, Booth CJ, Samulski JJ, Kafri T, McPhee SW, Samulski RJ.

Hum Gene Ther. 2015 Feb;26(2):69-81. doi: 10.1089/hum.2014.106.


The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

Finn JD, Nichols TC, Svoronos N, Merricks EP, Bellenger DA, Zhou S, Simioni P, High KA, Arruda VR.

Blood. 2012 Nov 29;120(23):4521-3. doi: 10.1182/blood-2012-06-440123.


Genetic modification of donor hepatocytes improves therapeutic efficacy for hemophilia B in mice.

Wu YM, Kao CY, Huang YJ, Yu IS, Lee HS, Lai HS, Lee PH, Lin CN, Lin SW.

Cell Transplant. 2010;19(9):1169-80. doi: 10.3727/096368910X503398.


Characterisation of factor IX with a glycine-to-valine missense mutation at residue 190 in a patient with severe haemophilia B.

Kao CY, Lin CN, Yang YL, Hamaguchi N, Yang SJ, Shen MC, Kao JT, Lin SW.

Thromb Haemost. 2011 Apr;105(4):616-26. doi: 10.1160/TH10-11-0762.


Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice.

Cantore A, Nair N, Della Valle P, Di Matteo M, Màtrai J, Sanvito F, Brombin C, Di Serio C, D'Angelo A, Chuah M, Naldini L, Vandendriessche T.

Blood. 2012 Nov 29;120(23):4517-20. doi: 10.1182/blood-2012-05-432591.


An important role for the activation peptide domain in controlling factor IX levels in the blood of haemophilia B mice.

Begbie ME, Mamdani A, Gataiance S, Eltringham-Smith LJ, Bhakta V, Hortelano G, Sheffield WP.

Thromb Haemost. 2005 Dec;94(6):1138-47.


Oral gene therapy for hemophilia B using chitosan-formulated FIX mutants.

Quade-Lyssy P, Milanov P, Abriss D, Ungerer C, Königs C, Seifried E, Schüttrumpf J.

J Thromb Haemost. 2014 Jun;12(6):932-42. doi: 10.1111/jth.12572.


Expression and Characterization of Gly-317 Variants of Factor IX Causing Variable Bleeding in Hemophilia B Patients.

Lu Q, Yang L, Manithody C, Wang X, Rezaie AR.

Biochemistry. 2015 Jun 23;54(24):3814-21. doi: 10.1021/acs.biochem.5b00270.


Changing residue 338 in human factor IX from arginine to alanine causes an increase in catalytic activity.

Chang J, Jin J, Lollar P, Bode W, Brandstetter H, Hamaguchi N, Straight DL, Stafford DW.

J Biol Chem. 1998 May 15;273(20):12089-94.


Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B.

Zhang R, Wang Q, Zhang L, Chen S.

Front Med. 2015 Mar;9(1):90-9. doi: 10.1007/s11684-015-0390-2.


Glycoengineered factor IX variants with improved pharmacokinetics and subcutaneous efficacy.

Brooks AR, Sim D, Gritzan U, Patel C, Blasko E, Feldman RI, Tang L, Ho E, Zhao XY, Apeler H, Murphy JE.

J Thromb Haemost. 2013 Sep;11(9):1699-706. doi: 10.1111/jth.12300.


Next generation FIX muteins with FVIII-independent activity for alternative treatment of hemophilia A.

Quade-Lyssy P, Abriss D, Milanov P, Ungerer C, Königs C, Seifried E, Schüttrumpf J.

J Thromb Haemost. 2014 Nov;12(11):1861-73. doi: 10.1111/jth.12731.


Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice.

Milanov P, Ivanciu L, Abriss D, Quade-Lyssy P, Miesbach W, Alesci S, Tonn T, Grez M, Seifried E, Schüttrumpf J.

Blood. 2012 Jan 12;119(2):602-11. doi: 10.1182/blood-2011-05-353672.


In vivo efficacy of human recombinant factor IX produced by the human hepatoma cell line HuH-7.

Enjolras N, Perot E, Le Quellec S, Indalecio A, Girard J, Negrier C, Dargaud Y.

Haemophilia. 2015 Jul;21(4):e317-21. doi: 10.1111/hae.12688.

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