172 related articles for article (PubMed ID: 19196177)
1. Bioengineered factor IX molecules with increased catalytic activity improve the therapeutic index of gene therapy vectors for hemophilia B.
Brunetti-Pierri N; Grove NC; Zuo Y; Edwards R; Palmer D; Cerullo V; Teruya J; Ng P
Hum Gene Ther; 2009 May; 20(5):479-85. PubMed ID: 19196177
[TBL] [Abstract][Full Text] [Related]
2. 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. PubMed ID: 25419787
[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. 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. PubMed ID: 24679056
[TBL] [Abstract][Full Text] [Related]
5. 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. PubMed ID: 20539913
[TBL] [Abstract][Full Text] [Related]
6. Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice.
Suwanmanee T; Hu G; Gui T; Bartholomae CC; Kutschera I; von Kalle C; Schmidt M; Monahan PE; Kafri T
Mol Ther; 2014 Mar; 22(3):567-574. PubMed ID: 23941813
[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. Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice.
Annoni A; Cantore A; Della Valle P; Goudy K; Akbarpour M; Russo F; Bartolaccini S; D'Angelo A; Roncarolo MG; Naldini L
EMBO Mol Med; 2013 Nov; 5(11):1684-97. PubMed ID: 24106222
[TBL] [Abstract][Full Text] [Related]
9. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA; Sullivan SK; Giermasz A; Rasko JEJ; Samelson-Jones BJ; Ducore J; Cuker A; Sullivan LM; Majumdar S; Teitel J; McGuinn CE; Ragni MV; Luk AY; Hui D; Wright JF; Chen Y; Liu Y; Wachtel K; Winters A; Tiefenbacher S; Arruda VR; van der Loo JCM; Zelenaia O; Takefman D; Carr ME; Couto LB; Anguela XM; High KA
N Engl J Med; 2017 Dec; 377(23):2215-2227. PubMed ID: 29211678
[TBL] [Abstract][Full Text] [Related]
10. Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose.
Wu Z; Sun J; Zhang T; Yin C; Yin F; Van Dyke T; Samulski RJ; Monahan PE
Mol Ther; 2008 Feb; 16(2):280-9. PubMed ID: 18059373
[TBL] [Abstract][Full Text] [Related]
11. Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B.
Harding TC; Koprivnikar KE; Tu GH; Zayek N; Lew S; Subramanian A; Sivakumaran A; Frey D; Ho K; VanRoey MJ; Nichols TC; Bellinger DA; Yendluri S; Waugh J; McArthur J; Veres G; Donahue BA
Gene Ther; 2004 Jan; 11(2):204-13. PubMed ID: 14712305
[TBL] [Abstract][Full Text] [Related]
12. 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]
13. 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]
14. Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.
Xu L; Gao C; Sands MS; Cai SR; Nichols TC; Bellinger DA; Raymer RA; McCorquodale S; Ponder KP
Blood; 2003 May; 101(10):3924-32. PubMed ID: 12531787
[TBL] [Abstract][Full Text] [Related]
15. 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]
16. A factor IX-deficient mouse model for hemophilia B gene therapy.
Wang L; Zoppè M; Hackeng TM; Griffin JH; Lee KF; Verma IM
Proc Natl Acad Sci U S A; 1997 Oct; 94(21):11563-6. PubMed ID: 9326649
[TBL] [Abstract][Full Text] [Related]
17. Balloon catheter delivery of helper-dependent adenoviral vector results in sustained, therapeutic hFIX expression in rhesus macaques.
Brunetti-Pierri N; Liou A; Patel P; Palmer D; Grove N; Finegold M; Piccolo P; Donnachie E; Rice K; Beaudet A; Mullins C; Ng P
Mol Ther; 2012 Oct; 20(10):1863-70. PubMed ID: 22828499
[TBL] [Abstract][Full Text] [Related]
18. 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]
19. 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. PubMed ID: 20492477
[TBL] [Abstract][Full Text] [Related]
20. 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]
[Next] [New Search]