270 related articles for article (PubMed ID: 25034357)
1. Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy.
Di Matteo M; Samara-Kuko E; Ward NJ; Waddington SN; McVey JH; Chuah MK; VandenDriessche T
Mol Ther; 2014 Sep; 22(9):1614-24. PubMed ID: 25034357
[TBL] [Abstract][Full Text] [Related]
2. 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]
3. 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]
4. 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]
5. 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]
6. Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo.
Mikkelsen JG; Yant SR; Meuse L; Huang Z; Xu H; Kay MA
Mol Ther; 2003 Oct; 8(4):654-65. PubMed ID: 14529839
[TBL] [Abstract][Full Text] [Related]
7. 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]
8. 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]
9. 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. PubMed ID: 25663062
[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. 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]
12. Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.
Hausl MA; Zhang W; Müther N; Rauschhuber C; Franck HG; Merricks EP; Nichols TC; Kay MA; Ehrhardt A
Mol Ther; 2010 Nov; 18(11):1896-906. PubMed ID: 20717103
[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. 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]
15. Development of adenovirus hybrid vectors for Sleeping Beauty transposition in large mammals.
Hausl M; Zhang W; Voigtländer R; Müther N; Rauschhuber C; Ehrhardt A
Curr Gene Ther; 2011 Oct; 11(5):363-74. PubMed ID: 21888620
[TBL] [Abstract][Full Text] [Related]
16. Hyperactive piggyBac gene transfer in human cells and in vivo.
Doherty JE; Huye LE; Yusa K; Zhou L; Craig NL; Wilson MH
Hum Gene Ther; 2012 Mar; 23(3):311-20. PubMed ID: 21992617
[TBL] [Abstract][Full Text] [Related]
17. A David promoter with Goliath strength.
Markusic DM; Herzog RW
Blood; 2014 May; 123(20):3068-9. PubMed ID: 24832942
[TBL] [Abstract][Full Text] [Related]
18. 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]
19. Transposons: Moving Forward from Preclinical Studies to Clinical Trials.
Tipanee J; VandenDriessche T; Chuah MK
Hum Gene Ther; 2017 Nov; 28(11):1087-1104. PubMed ID: 28920716
[TBL] [Abstract][Full Text] [Related]
20. Vector modifications to eliminate transposase expression following piggyBac-mediated transgenesis.
Chakraborty S; Ji H; Chen J; Gersbach CA; Leong KW
Sci Rep; 2014 Dec; 4():7403. PubMed ID: 25492703
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
