504 related articles for article (PubMed ID: 18059373)
21. Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo.
Miao CH; Thompson AR; Loeb K; Ye X
Mol Ther; 2001 Jun; 3(6):947-57. PubMed ID: 11407909
[TBL] [Abstract][Full Text] [Related]
22. Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.
Nair N; Rincon MY; Evens H; Sarcar S; Dastidar S; Samara-Kuko E; Ghandeharian O; Man Viecelli H; Thöny B; De Bleser P; VandenDriessche T; Chuah MK
Blood; 2014 May; 123(20):3195-9. PubMed ID: 24637359
[TBL] [Abstract][Full Text] [Related]
23. Potential limits of AAV-based gene therapy with the use of new transgenes expressing factor IX fusion proteins.
Le Quellec S; Dane A; Enjolras N; McIntosh J; Rosales C; Negrier C; Nathwani A
Haemophilia; 2019 Jan; 25(1):e11-e18. PubMed ID: 30520547
[TBL] [Abstract][Full Text] [Related]
24. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver.
Wang L; Nichols TC; Read MS; Bellinger DA; Verma IM
Mol Ther; 2000 Feb; 1(2):154-8. PubMed ID: 10933925
[TBL] [Abstract][Full Text] [Related]
25. AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects.
Hasbrouck NC; High KA
Gene Ther; 2008 Jun; 15(11):870-5. PubMed ID: 18432276
[TBL] [Abstract][Full Text] [Related]
26. 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]
27. 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]
28. Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep.
David AL; McIntosh J; Peebles DM; Cook T; Waddington S; Weisz B; Wigley V; Abi-Nader K; Boyd M; Davidoff AM; Nathwani AC
Hum Gene Ther; 2011 Apr; 22(4):419-26. PubMed ID: 20919876
[TBL] [Abstract][Full Text] [Related]
29. Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.
Markusic DM; Nichols TC; Merricks EP; Palaschak B; Zolotukhin I; Marsic D; Zolotukhin S; Srivastava A; Herzog RW
J Transl Med; 2017 May; 15(1):94. PubMed ID: 28460646
[TBL] [Abstract][Full Text] [Related]
30. 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]
31. Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy.
Niemeyer GP; Herzog RW; Mount J; Arruda VR; Tillson DM; Hathcock J; van Ginkel FW; High KA; Lothrop CD
Blood; 2009 Jan; 113(4):797-806. PubMed ID: 18957684
[TBL] [Abstract][Full Text] [Related]
32. Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy.
Herzog RW; Fields PA; Arruda VR; Brubaker JO; Armstrong E; McClintock D; Bellinger DA; Couto LB; Nichols TC; High KA
Hum Gene Ther; 2002 Jul; 13(11):1281-91. PubMed ID: 12162811
[TBL] [Abstract][Full Text] [Related]
33. [Preparation of rAAV2/hFIX and experimentally application to gene therapy for hemophilia B].
Peng JQ; Dong XY; Peng M; Chen L; Tan SP; Yuan H; Chen FP; Xue JL; Wu XB
Zhonghua Xue Ye Xue Za Zhi; 2004 Sep; 25(9):513-8. PubMed ID: 15569526
[TBL] [Abstract][Full Text] [Related]
34. Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector.
Liu YL; Mingozzi F; Rodriguéz-Colôn SM; Joseph S; Dobrzynski E; Suzuki T; High KA; Herzog RW
Hum Gene Ther; 2004 Aug; 15(8):783-92. PubMed ID: 15319035
[TBL] [Abstract][Full Text] [Related]
35. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Nathwani AC; Tuddenham EG; Rangarajan S; Rosales C; McIntosh J; Linch DC; Chowdary P; Riddell A; Pie AJ; Harrington C; O'Beirne J; Smith K; Pasi J; Glader B; Rustagi P; Ng CY; Kay MA; Zhou J; Spence Y; Morton CL; Allay J; Coleman J; Sleep S; Cunningham JM; Srivastava D; Basner-Tschakarjan E; Mingozzi F; High KA; Gray JT; Reiss UM; Nienhuis AW; Davidoff AM
N Engl J Med; 2011 Dec; 365(25):2357-65. PubMed ID: 22149959
[TBL] [Abstract][Full Text] [Related]
36. New and improved AAVenues: current status of hemophilia B gene therapy.
Brimble MA; Reiss UM; Nathwani AC; Davidoff AM
Expert Opin Biol Ther; 2016; 16(1):79-92. PubMed ID: 26524468
[TBL] [Abstract][Full Text] [Related]
37. Theodore E. Woodward Award. AAV-mediated gene transfer for hemophilia.
High KA
Trans Am Clin Climatol Assoc; 2003; 114():337-51; discussion 351-2. PubMed ID: 12813929
[TBL] [Abstract][Full Text] [Related]
38. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
Arruda VR; Stedman HH; Haurigot V; Buchlis G; Baila S; Favaro P; Chen Y; Franck HG; Zhou S; Wright JF; Couto LB; Jiang H; Pierce GF; Bellinger DA; Mingozzi F; Nichols TC; High KA
Blood; 2010 Jun; 115(23):4678-88. PubMed ID: 20335222
[TBL] [Abstract][Full Text] [Related]
39. Gene therapy for hemophilia: advancing beyond the first clinical success.
Monahan PE; Gui T
Curr Opin Hematol; 2013 Sep; 20(5):410-6. PubMed ID: 23852185
[TBL] [Abstract][Full Text] [Related]
40. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.
Jiang H; Pierce GF; Ozelo MC; de Paula EV; Vargas JA; Smith P; Sommer J; Luk A; Manno CS; High KA; Arruda VR
Mol Ther; 2006 Sep; 14(3):452-5. PubMed ID: 16822719
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
