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Items: 1 to 20 of 355

1.

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.

2.

Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application.

Monahan PE, Lothrop CD, Sun J, Hirsch ML, Kafri T, Kantor B, Sarkar R, Tillson DM, Elia JR, Samulski RJ.

Mol Ther. 2010 Nov;18(11):1907-16. doi: 10.1038/mt.2010.170. Epub 2010 Aug 10.

3.

Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction.

Finn JD, Hui D, Downey HD, Dunn D, Pien GC, Mingozzi F, Zhou S, High KA.

Mol Ther. 2010 Jan;18(1):135-42. doi: 10.1038/mt.2009.257. Epub 2009 Nov 10.

4.

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.

5.

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine.

McIntosh JH, Cochrane M, Cobbold S, Waldmann H, Nathwani SA, Davidoff AM, Nathwani AC.

Gene Ther. 2012 Jan;19(1):78-85. doi: 10.1038/gt.2011.64. Epub 2011 Jun 30.

6.

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.

7.

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.

8.
9.

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver.

Martino AT, Suzuki M, Markusic DM, Zolotukhin I, Ryals RC, Moghimi B, Ertl HC, Muruve DA, Lee B, Herzog RW.

Blood. 2011 Jun 16;117(24):6459-68. doi: 10.1182/blood-2010-10-314518. Epub 2011 Apr 7.

10.

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.

11.

Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: therapeutic expression of factor IX at reduced vector doses.

Jayandharan GR, Zhong L, Sack BK, Rivers AE, Li M, Li B, Herzog RW, Srivastava A.

Hum Gene Ther. 2010 Mar;21(3):271-83. doi: 10.1089/hum.2009.100.

12.

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.

13.
14.

Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model.

Favaro P, Finn JD, Siner JI, Wright JF, High KA, Arruda VR.

Hum Gene Ther. 2011 Jul;22(7):843-52. doi: 10.1089/hum.2010.155. Epub 2011 Mar 8.

15.

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. doi: 10.1089/hum.2010.007. Epub 2011 Feb 2.

PMID:
20919876
16.

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.

17.

Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector.

Mingozzi F, Schüttrumpf J, Arruda VR, Liu Y, Liu YL, High KA, Xiao W, Herzog RW.

J Virol. 2002 Oct;76(20):10497-502.

18.

Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes.

Dane AP, Wowro SJ, Cunningham SC, Alexander IE.

Gene Ther. 2013 Apr;20(4):460-4. doi: 10.1038/gt.2012.67. Epub 2012 Aug 16.

PMID:
22895507
19.

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.

20.

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.

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