Nienhuis AW - Search Results

1

Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors.

Koldej RM, Carney G, Wielgosz MM, Zhou S, Zhan J, Sorrentino BP, Nienhuis AW. Koldej RM, et al. Among authors: nienhuis aw. Hum Gene Ther Clin Dev. 2013 Jun;24(2):77-85. doi: 10.1089/humc.2012.244. Epub 2013 Jun 20. Hum Gene Ther Clin Dev. 2013. PMID: 23786330 Free PMC article.

2

Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector.

Singh S, Khan I, Khim S, Seymour B, Sommer K, Wielgosz M, Norgaard Z, Kiem HP, Adair J, Liggitt D, Nienhuis A, Rawlings DJ. Singh S, et al. Mol Ther Methods Clin Dev. 2016 Dec 18;4:1-16. doi: 10.1016/j.omtm.2016.11.001. eCollection 2017 Mar 17. Mol Ther Methods Clin Dev. 2016. PMID: 28344987 Free PMC article.

3

Suppression of clonal dominance in cultured human lymphoid cells by addition of the cHS4 insulator to a lentiviral vector.

Evans-Galea MV, Wielgosz MM, Hanawa H, Srivastava DK, Nienhuis AW. Evans-Galea MV, et al. Among authors: nienhuis aw. Mol Ther. 2007 Apr;15(4):801-9. doi: 10.1038/sj.mt.6300103. Epub 2007 Feb 13. Mol Ther. 2007. PMID: 17299406 Free article.

4

Transduction of human primitive repopulating hematopoietic cells with lentiviral vectors pseudotyped with various envelope proteins.

Kim YS, Wielgosz MM, Hargrove P, Kepes S, Gray J, Persons DA, Nienhuis AW. Kim YS, et al. Among authors: nienhuis aw. Mol Ther. 2010 Jul;18(7):1310-7. doi: 10.1038/mt.2010.48. Epub 2010 Apr 6. Mol Ther. 2010. PMID: 20372106 Free PMC article.

5

Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy.

Wielgosz MM, Kim YS, Carney GG, Zhan J, Reddivari M, Coop T, Heath RJ, Brown SA, Nienhuis AW. Wielgosz MM, et al. Among authors: nienhuis aw. Mol Ther Methods Clin Dev. 2015 Jan 21;2:14063. doi: 10.1038/mtm.2014.63. eCollection 2015. Mol Ther Methods Clin Dev. 2015. PMID: 26052531 Free PMC article.

6

The identification of hematopoietic-specific regulatory elements for WASp gene expression.

Zhan J, Johnson IM, Wielgosz M, Nienhuis AW. Zhan J, et al. Among authors: nienhuis aw. Mol Ther Methods Clin Dev. 2016 Dec 14;3:16077. doi: 10.1038/mtm.2016.77. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 28035317 Free PMC article. Review.

7

Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells.

Persons DA, Allay ER, Sawai N, Hargrove PW, Brent TP, Hanawa H, Nienhuis AW, Sorrentino BP. Persons DA, et al. Among authors: nienhuis aw. Blood. 2003 Jul 15;102(2):506-13. doi: 10.1182/blood-2003-03-0677. Epub 2003 Mar 27. Blood. 2003. PMID: 12663444 Free article.

8

Genotoxicity of retroviral integration in hematopoietic cells.

Nienhuis AW, Dunbar CE, Sorrentino BP. Nienhuis AW, et al. Mol Ther. 2006 Jun;13(6):1031-49. doi: 10.1016/j.ymthe.2006.03.001. Epub 2006 Apr 19. Mol Ther. 2006. PMID: 16624621 Free article. Review.

9

Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial.

Allay JA, Sleep S, Long S, Tillman DM, Clark R, Carney G, Fagone P, McIntosh JH, Nienhuis AW, Davidoff AM, Nathwani AC, Gray JT. Allay JA, et al. Among authors: nienhuis aw. Hum Gene Ther. 2011 May;22(5):595-604. doi: 10.1089/hum.2010.202. Hum Gene Ther. 2011. PMID: 21410419 Free PMC article.

10

Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.

Zhao HF, Abraham A, Kim YS, Wang YD, Pestina T, Zhan J, Humphries K, Nienhuis AW, Persons DA. Zhao HF, et al. Among authors: nienhuis aw. Mol Ther. 2017 Mar 1;25(3):593-605. doi: 10.1016/j.ymthe.2017.01.019. Epub 2017 Feb 9. Mol Ther. 2017. PMID: 28190779 Free PMC article.

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