BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

176 related articles for article (PubMed ID: 33764159)

  • 1. Treatment of a Hemophilia B Mouse Model with Platelet-Targeted Expression of Factor IX Padua.
    Li B; Wu Z; Xu W; Han W; Liu J; Wang D; Zhang G
    Hum Gene Ther; 2021 May; 32(9-10):506-516. PubMed ID: 33764159
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice.
    Zhang G; Shi Q; Fahs SA; Kuether EL; Walsh CE; Montgomery RR
    Blood; 2010 Aug; 116(8):1235-43. PubMed ID: 20445020
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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; Arruda VR
    Blood; 2015 Mar; 125(10):1553-61. PubMed ID: 25568350
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua.
    Robinson MM; George LA; Carr ME; Samelson-Jones BJ; Arruda VR; Murphy JE; Rybin D; Rupon J; High KA; Tiefenbacher S
    J Thromb Haemost; 2021 May; 19(5):1212-1218. PubMed ID: 33636038
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Activated factor X targeted stored in platelets as an effective gene therapy strategy for both hemophilia A and B.
    Wang D; Shao X; Wang Q; Pan X; Dai Y; Yao S; Yin T; Wang Z; Zhu J; Xi X; Chen Z; Chen S; Zhang G
    Clin Transl Med; 2021 Mar; 11(3):e375. PubMed ID: 33783994
    [TBL] [Abstract][Full Text] [Related]  

  • 6. CRISPR-Mediated In Situ Introduction or Integration of
    Tang Q; Hu Z; Zhao J; Zhou T; Tang S; Wang P; Xiao R; Chen Y; Wu L; Zhou M; Liang D
    Int J Mol Sci; 2023 May; 24(10):. PubMed ID: 37240366
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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. PubMed ID: 23676890
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua.
    Nair N; De Wolf D; Nguyen PA; Pham QH; Samara-Kuko E; Landau J; Blouse GE; Chuah MK; VandenDriessche T
    Blood; 2021 May; 137(21):2902-2906. PubMed ID: 33735915
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with preexisting anti-FIX immunity.
    Schroeder JA; Chen J; Chen Y; Cai Y; Yu H; Mattson JG; Monahan PE; Shi Q
    Blood Adv; 2021 Mar; 5(5):1224-1238. PubMed ID: 33646304
    [TBL] [Abstract][Full Text] [Related]  

  • 10. 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; 120(23):4521-3. PubMed ID: 22919027
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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. PubMed ID: 25795563
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Fusion of engineered albumin with factor IX Padua extends half-life and improves coagulant activity.
    Lombardi S; Aaen KH; Nilsen J; Ferrarese M; Gjølberg TT; Bernardi F; Pinotti M; Andersen JT; Branchini A
    Br J Haematol; 2021 Jul; 194(2):453-462. PubMed ID: 34109608
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice.
    Chen Y; Schroeder JA; Kuether EL; Zhang G; Shi Q
    Mol Ther; 2014 Jan; 22(1):169-77. PubMed ID: 24042561
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Characteristics of FXa-storing platelets in hemophilia B mice and the influence of alcohol on the platelets.
    Han W; Huang R; Li B; Liu L; Xu W; Zhang G
    Sci Rep; 2023 Oct; 13(1):16488. PubMed ID: 37779119
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Etranacogene dezaparvovec-drlb gene therapy for patients with hemophilia B (congenital factor IX deficiency).
    Sekayan T; Simmons DH; von Drygalski A
    Expert Opin Biol Ther; 2023; 23(12):1173-1184. PubMed ID: 37962325
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B.
    Shah J; Kim H; Sivamurthy K; Monahan PE; Fries M
    Curr Med Res Opin; 2023 Feb; 39(2):227-237. PubMed ID: 36285399
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice.
    Le Quellec S; Dane AP; Barbon E; Bordet JC; Mingozzi F; Dargaud Y; Marais T; Biferi MG; Négrier C; Nathawani AC; Enjolras N
    Thromb Haemost; 2019 Dec; 119(12):1956-1967. PubMed ID: 31659733
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity.
    Samelson-Jones BJ; Finn JD; George LA; Camire RM; Arruda VR
    JCI Insight; 2019 Jun; 5(14):. PubMed ID: 31219805
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Hemophilia Gene Therapy: Ready for Prime Time?
    VandenDriessche T; Chuah MK
    Hum Gene Ther; 2017 Nov; 28(11):1013-1023. PubMed ID: 28793786
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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; 120(23):4517-20. PubMed ID: 23043073
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.