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Items: 1 to 20 of 109

1.

Gene therapy for primary immunodeficiencies: looking ahead, toward gene correction.

Pessach IM, Notarangelo LD.

J Allergy Clin Immunol. 2011 Jun;127(6):1344-50. doi: 10.1016/j.jaci.2011.02.027. Epub 2011 Mar 25. Review.

2.

Gene therapy for primary immunodeficiencies: Part 2.

Aiuti A, Bacchetta R, Seger R, Villa A, Cavazzana-Calvo M.

Curr Opin Immunol. 2012 Oct;24(5):585-91. doi: 10.1016/j.coi.2012.07.012. Epub 2012 Aug 18. Review.

PMID:
22909900
3.

Gene therapy for PIDs: progress, pitfalls and prospects.

Mukherjee S, Thrasher AJ.

Gene. 2013 Aug 10;525(2):174-81. doi: 10.1016/j.gene.2013.03.098. Epub 2013 Apr 6. Review.

4.

Gene therapy for primary immunodeficiencies.

Kildebeck E, Checketts J, Porteus M.

Curr Opin Pediatr. 2012 Dec;24(6):731-8. doi: 10.1097/MOP.0b013e328359e480. Review.

PMID:
23073463
5.

Gene therapy for primary immunodeficiencies.

Rivat C, Santilli G, Gaspar HB, Thrasher AJ.

Hum Gene Ther. 2012 Jul;23(7):668-75. doi: 10.1089/hum.2012.116. Review.

6.

Gene therapy for primary immunodeficiencies: Part 1.

Cavazzana-Calvo M, Fischer A, Hacein-Bey-Abina S, Aiuti A.

Curr Opin Immunol. 2012 Oct;24(5):580-4. doi: 10.1016/j.coi.2012.08.008. Epub 2012 Sep 12. Review.

PMID:
22981681
7.

Update on gene therapy for immunodeficiencies.

Kohn DB.

Clin Immunol. 2010 May;135(2):247-54. doi: 10.1016/j.clim.2009.12.003. Epub 2010 Jan 13. Review.

8.

Progress and prospects: zinc-finger nucleases as gene therapy agents.

Carroll D.

Gene Ther. 2008 Nov;15(22):1463-8. doi: 10.1038/gt.2008.145. Epub 2008 Sep 11. Review.

9.

Gene repair of an Usher syndrome causing mutation by zinc-finger nuclease mediated homologous recombination.

Overlack N, Goldmann T, Wolfrum U, Nagel-Wolfrum K.

Invest Ophthalmol Vis Sci. 2012 Jun 26;53(7):4140-6. doi: 10.1167/iovs.12-9812.

PMID:
22661463
10.

Zinc-finger nuclease-induced gene repair with oligodeoxynucleotides: wanted and unwanted target locus modifications.

Radecke S, Radecke F, Cathomen T, Schwarz K.

Mol Ther. 2010 Apr;18(4):743-53. doi: 10.1038/mt.2009.304. Epub 2010 Jan 12.

11.

Gene therapy for primary immunodeficiencies: current status and future prospects.

Qasim W, Gennery AR.

Drugs. 2014 Jun;74(9):963-9. doi: 10.1007/s40265-014-0223-7. Review.

PMID:
24848753
12.

Current progress on gene therapy for primary immunodeficiencies.

Zhang L, Thrasher AJ, Gaspar HB.

Gene Ther. 2013 Oct;20(10):963-9. doi: 10.1038/gt.2013.21. Epub 2013 May 30. Review.

PMID:
23719067
13.

In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases.

Sebastiano V, Maeder ML, Angstman JF, Haddad B, Khayter C, Yeo DT, Goodwin MJ, Hawkins JS, Ramirez CL, Batista LF, Artandi SE, Wernig M, Joung JK.

Stem Cells. 2011 Nov;29(11):1717-26. doi: 10.1002/stem.718.

14.

Biological and biomedical applications of engineered nucleases.

Pan Y, Xiao L, Li AS, Zhang X, Sirois P, Zhang J, Li K.

Mol Biotechnol. 2013 Sep;55(1):54-62. doi: 10.1007/s12033-012-9613-9. Review.

PMID:
23089945
15.

Targeted transgene insertion into the AAVS1 locus driven by baculoviral vector-mediated zinc finger nuclease expression in human-induced pluripotent stem cells.

Tay FC, Tan WK, Goh SL, Ramachandra CJ, Lau CH, Zhu H, Chen C, Du S, Phang RZ, Shahbazi M, Fan W, Wang S.

J Gene Med. 2013 Oct;15(10):384-95. doi: 10.1002/jgm.2745.

PMID:
24105820
16.

Zinc-finger nucleases for somatic gene therapy: the next frontier.

Rahman SH, Maeder ML, Joung JK, Cathomen T.

Hum Gene Ther. 2011 Aug;22(8):925-33. doi: 10.1089/hum.2011.087. Epub 2011 Jul 22. Review.

17.

Targeted mutagenesis in Arabidopsis using zinc-finger nucleases.

Zhang F, Voytas DF.

Methods Mol Biol. 2011;701:167-77. doi: 10.1007/978-1-61737-957-4_9.

PMID:
21181530
18.

Designing and testing chimeric zinc finger transposases.

Wilson MH, George AL Jr.

Methods Mol Biol. 2010;649:353-63. doi: 10.1007/978-1-60761-753-2_22.

PMID:
20680846
19.

Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination.

Coluccio A, Miselli F, Lombardo A, Marconi A, Malagoli Tagliazucchi G, Gonçalves MA, Pincelli C, Maruggi G, Del Rio M, Naldini L, Larcher F, Mavilio F, Recchia A.

Mol Ther. 2013 Sep;21(9):1695-704. doi: 10.1038/mt.2013.143. Epub 2013 Jun 13.

20.

Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme.

Wang J, Friedman G, Doyon Y, Wang NS, Li CJ, Miller JC, Hua KL, Yan JJ, Babiarz JE, Gregory PD, Holmes MC.

Genome Res. 2012 Jul;22(7):1316-26. doi: 10.1101/gr.122879.111. Epub 2012 Mar 20.

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