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Items: 1 to 20 of 103

1.

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates.

Nathwani AC, Gray JT, McIntosh J, Ng CY, Zhou J, Spence Y, Cochrane M, Gray E, Tuddenham EG, Davidoff AM.

Blood. 2007 Feb 15;109(4):1414-21. Epub 2006 Nov 7.

2.

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.

Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN, Tuddenham EG, Kemball-Cook G, McIntosh J, Boon-Spijker M, Mertens K, Davidoff AM.

Blood. 2006 Apr 1;107(7):2653-61. Epub 2005 Dec 1.

3.

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.

4.

Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.

Mattar CN, Nathwani AC, Waddington SN, Dighe N, Kaeppel C, Nowrouzi A, Mcintosh J, Johana NB, Ogden B, Fisk NM, Davidoff AM, David A, Peebles D, Valentine MB, Appelt JU, von Kalle C, Schmidt M, Biswas A, Choolani M, Chan JK.

Mol Ther. 2011 Nov;19(11):1950-60. doi: 10.1038/mt.2011.107. Epub 2011 May 31.

5.

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
6.

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.

7.

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.

8.

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 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10.

9.

Enhancing transduction of the liver by adeno-associated viral vectors.

Nathwani AC, Cochrane M, McIntosh J, Ng CY, Zhou J, Gray JT, Davidoff AM.

Gene Ther. 2009 Jan;16(1):60-9. doi: 10.1038/gt.2008.137. Epub 2008 Aug 14.

10.

Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy.

Grimm D, Zhou S, Nakai H, Thomas CE, Storm TA, Fuess S, Matsushita T, Allen J, Surosky R, Lochrie M, Meuse L, McClelland A, Colosi P, Kay MA.

Blood. 2003 Oct 1;102(7):2412-9. Epub 2003 Jun 5.

11.

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.

12.

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
13.

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.

Nathwani AC, Davidoff A, Hanawa H, Zhou JF, Vanin EF, Nienhuis AW.

Blood. 2001 Mar 1;97(5):1258-65.

14.

Gene therapy for hemophilia B mice with scAAV8-LP1-hFIX.

Lu W, Zhou Q, Yang H, Wang H, Gu Y, Shen Q, Xue J, Dong X, Chen J.

Front Med. 2016 Jun;10(2):212-8. doi: 10.1007/s11684-016-0438-y. Epub 2016 Apr 6.

PMID:
27052253
15.

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy.

Vandendriessche T, Thorrez L, Acosta-Sanchez A, Petrus I, Wang L, Ma L, DE Waele L, Iwasaki Y, Gillijns V, Wilson JM, Collen D, Chuah MK.

J Thromb Haemost. 2007 Jan;5(1):16-24. Epub 2006 Sep 26.

16.

Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver.

Ohashi K, Nakai H, Couto LB, Kay MA.

Hum Gene Ther. 2005 Mar;16(3):299-306.

PMID:
15812225
18.

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.

19.
20.

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.

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