187 related articles for article (PubMed ID: 24679056)
21. CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout mice.
Wang L; Yang Y; Breton CA; White J; Zhang J; Che Y; Saveliev A; McMenamin D; He Z; Latshaw C; Li M; Wilson JM
Blood; 2019 Jun; 133(26):2745-2752. PubMed ID: 30975639
[TBL] [Abstract][Full Text] [Related]
22. Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system.
DeRosa F; Guild B; Karve S; Smith L; Love K; Dorkin JR; Kauffman KJ; Zhang J; Yahalom B; Anderson DG; Heartlein MW
Gene Ther; 2016 Oct; 23(10):699-707. PubMed ID: 27356951
[TBL] [Abstract][Full Text] [Related]
23. 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. PubMed ID: 23692404
[TBL] [Abstract][Full Text] [Related]
24. 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]
25. 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]
26. 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]
27. Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression.
Fang B; Eisensmith RC; Wang H; Kay MA; Cross RE; Landen CN; Gordon G; Bellinger DA; Read MS; Hu PC
Hum Gene Ther; 1995 Aug; 6(8):1039-44. PubMed ID: 7578416
[TBL] [Abstract][Full Text] [Related]
28. 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]
29. 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]
30. 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]
31. 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]
32. Dexamethasone Transiently Enhances Transgene Expression in the Liver When Administered at Late-Phase Post Long-Term Adeno-Associated Virus Transduction.
Chai Z; Zhang X; Dobbins AL; Samulski RJ; Merricks EP; Nichols TC; Li C
Hum Gene Ther; 2022 Feb; 33(3-4):119-130. PubMed ID: 34617445
[TBL] [Abstract][Full Text] [Related]
33. Factor IX variants improve gene therapy efficacy for hemophilia B.
Schuettrumpf J; Herzog RW; Schlachterman A; Kaufhold A; Stafford DW; Arruda VR
Blood; 2005 Mar; 105(6):2316-23. PubMed ID: 15550487
[TBL] [Abstract][Full Text] [Related]
34. Efficient induction of immune tolerance to coagulation factor IX following direct intramuscular gene transfer.
Cohn EF; Zhuo J; Kelly ME; Chao HJ
J Thromb Haemost; 2007 Jun; 5(6):1227-36. PubMed ID: 17362228
[TBL] [Abstract][Full Text] [Related]
35. 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]
36. Long-term phenotypic correction in factor IX knockout mice by using ΦC31 integrase-mediated gene therapy.
Keravala A; Chavez CL; Hu G; Woodard LE; Monahan PE; Calos MP
Gene Ther; 2011 Aug; 18(8):842-8. PubMed ID: 21412285
[TBL] [Abstract][Full Text] [Related]
37. [Non-viral gene transfer results in therapeutic factor IX levels in haemophilia B mice].
Schüttrumpf J; Milanov P; Roth S; Seifried E; Tonn T
Hamostaseologie; 2008 Oct; 28 Suppl 1():S92-5. PubMed ID: 18958345
[TBL] [Abstract][Full Text] [Related]
38. 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]
39. 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]
40. Complete and sustained phenotypic correction of hemophilia B in mice following hepatic gene transfer of a high-expressing human factor IX plasmid.
Ye X; Loeb KR; Stafford DW; Thompson AR; Miao CH
J Thromb Haemost; 2003 Jan; 1(1):103-11. PubMed ID: 12871546
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
