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Items: 1 to 20 of 369

1.

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. doi: 10.1089/hum.2014.106. Epub 2015 Jan 21.

2.

Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX.

Sun J, Hakobyan N, Valentino LA, Feldman BL, Samulski RJ, Monahan PE.

Blood. 2008 Dec 1;112(12):4532-41. doi: 10.1182/blood-2008-01-131417. Epub 2008 Aug 20.

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 Jr, Arruda VR.

Blood. 2015 Mar 5;125(10):1553-61. doi: 10.1182/blood-2014-07-588194. Epub 2015 Jan 7.

4.

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.

Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, Zhou S, Scallan CD, Sommer J, Vijay S, Mingozzi F, High KA, Pierce GF.

Blood. 2006 Nov 15;108(10):3321-8. Epub 2006 Jul 25.

5.

Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo.

Sen D, Gadkari RA, Sudha G, Gabriel N, Kumar YS, Selot R, Samuel R, Rajalingam S, Ramya V, Nair SC, Srinivasan N, Srivastava A, Jayandharan GR.

Hum Gene Ther Methods. 2013 Apr;24(2):104-16. doi: 10.1089/hgtb.2012.195.

6.

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. doi: 10.1160/TH09-11-0792. Epub 2010 Jun 10.

PMID:
20539913
7.

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 29;120(23):4521-3. doi: 10.1182/blood-2012-06-440123. Epub 2012 Aug 23.

8.

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. doi: 10.1160/TH13-02-0154. Epub 2013 May 16.

PMID:
23676890
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. doi: 10.1007/s11684-015-0390-2. Epub 2015 Feb 7.

PMID:
25663062
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. Epub 2007 Dec 4.

11.

Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes.

Li H, Murphy SL, Giles-Davis W, Edmonson S, Xiang Z, Li Y, Lasaro MO, High KA, Ertl HC.

Mol Ther. 2007 Apr;15(4):792-800. Epub 2007 Jan 23.

12.

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 10;115(23):4678-88. doi: 10.1182/blood-2009-12-261156. Epub 2010 Mar 24.

13.

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 1;15(1):94. doi: 10.1186/s12967-017-1200-1.

14.

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

Arruda VR, Schuettrumpf J, Herzog RW, Nichols TC, Robinson N, Lotfi Y, Mingozzi F, Xiao W, Couto LB, High KA.

Blood. 2004 Jan 1;103(1):85-92. Epub 2003 Sep 11.

15.

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM.

Mol Ther. 2011 May;19(5):876-85. doi: 10.1038/mt.2010.274. Epub 2011 Jan 18.

16.

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.

PMID:
14712305
17.

Gene therapy for hemophilia: advancing beyond the first clinical success.

Monahan PE, Gui T.

Curr Opin Hematol. 2013 Sep;20(5):410-6. doi: 10.1097/MOH.0b013e328363c1a1. Review.

PMID:
23852185
18.

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 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.

19.
20.

Theodore E. Woodward Award. AAV-mediated gene transfer for hemophilia.

High KA.

Trans Am Clin Climatol Assoc. 2003;114:337-51; discussion 351-2. Review.

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