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Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells.

Chang CJ, Bouhassira EE.

Blood. 2012 Nov 8;120(19):3906-14. doi: 10.1182/blood-2012-03-420703.


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.


Factor-induced Reprogramming and Zinc Finger Nuclease-aided Gene Targeting Cause Different Genome Instability in β-Thalassemia Induced Pluripotent Stem Cells (iPSCs).

Ma N, Shan Y, Liao B, Kong G, Wang C, Huang K, Zhang H, Cai X, Chen S, Pei D, Chen N, Pan G.

J Biol Chem. 2015 May 8;290(19):12079-89. doi: 10.1074/jbc.M114.624999.


Zinc finger nuclease-expressing baculoviral vectors mediate targeted genome integration of reprogramming factor genes to facilitate the generation of human induced pluripotent stem cells.

Phang RZ, Tay FC, Goh SL, Lau CH, Zhu H, Tan WK, Liang Q, Chen C, Du S, Li Z, Tay JC, Wu C, Zeng J, Fan W, Toh HC, Wang S.

Stem Cells Transl Med. 2013 Dec;2(12):935-45. doi: 10.5966/sctm.2013-0043.


Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free β-thalassemia induced pluripotent stem cells.

Ma N, Liao B, Zhang H, Wang L, Shan Y, Xue Y, Huang K, Chen S, Zhou X, Chen Y, Pei D, Pan G.

J Biol Chem. 2013 Nov 29;288(48):34671-9. doi: 10.1074/jbc.M113.496174.


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.


Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells.

Papapetrou EP, Lee G, Malani N, Setty M, Riviere I, Tirunagari LM, Kadota K, Roth SL, Giardina P, Viale A, Leslie C, Bushman FD, Studer L, Sadelain M.

Nat Biotechnol. 2011 Jan;29(1):73-8. doi: 10.1038/nbt.1717.


A Doxycycline-Inducible System for Genetic Correction of iPSC Disease Models.

Sim X, Cardenas-Diaz FL, French DL, Gadue P.

Methods Mol Biol. 2016;1353:13-23. doi: 10.1007/7651_2014_179.


Chromatin structure of two genomic sites for targeted transgene integration in induced pluripotent stem cells and hematopoietic stem cells.

van Rensburg R, Beyer I, Yao XY, Wang H, Denisenko O, Li ZY, Russell DW, Miller DG, Gregory P, Holmes M, Bomsztyk K, Lieber A.

Gene Ther. 2013 Feb;20(2):201-14. doi: 10.1038/gt.2012.25.


alpha-Thalassemia-like globin gene expression by primitive erythrocytes derived from human embryonic stem cells.

Honig GR, Lu SJ, Feng Q, Vida LN, Lee BS, Lanza R.

Hemoglobin. 2010 Jan;34(2):145-50. doi: 10.3109/03630261003676850.


TALEN-mediated generation and genetic correction of disease-specific human induced pluripotent stem cells.

Ramalingam S, Annaluru N, Kandavelou K, Chandrasegaran S.

Curr Gene Ther. 2014;14(6):461-72.


Genome editing in induced pluripotent stem cells.

Cheng LT, Sun LT, Tada T.

Genes Cells. 2012 Jun;17(6):431-8. doi: 10.1111/j.1365-2443.2012.01599.x. Review.


A stepwise α-thalassemia screening strategy in high-prevalence areas.

Alkindi SS, Alzadjali S, Daar S, Sindhuvi E, Wali Y, Pathare AV, Venugopal S, Lapoumeroulie C, Srivastava A, Krishnamoorthy R.

Eur J Haematol. 2013 Aug;91(2):164-9. doi: 10.1111/ejh.12136.


Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging.

Wang Y, Zhang WY, Hu S, Lan F, Lee AS, Huber B, Lisowski L, Liang P, Huang M, de Almeida PE, Won JH, Sun N, Robbins RC, Kay MA, Urnov FD, Wu JC.

Circ Res. 2012 Dec 7;111(12):1494-503. doi: 10.1161/CIRCRESAHA.112.274969.


The molecular basis of alpha-thalassemia in Thailand.

Winichagoon P, Fucharoen S, Wasi P.

Southeast Asian J Trop Med Public Health. 1992;23 Suppl 2:7-13.


A zinc-finger transcriptional activator designed to interact with the gamma-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts.

Wilber A, Tschulena U, Hargrove PW, Kim YS, Persons DA, Barbas CF 3rd, Nienhuis AW.

Blood. 2010 Apr 15;115(15):3033-41. doi: 10.1182/blood-2009-08-240556.


Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice.

Wang Y, Zheng CG, Jiang Y, Zhang J, Chen J, Yao C, Zhao Q, Liu S, Chen K, Du J, Yang Z, Gao S.

Cell Res. 2012 Apr;22(4):637-48. doi: 10.1038/cr.2012.23.


Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.

Xie F, Ye L, Chang JC, Beyer AI, Wang J, Muench MO, Kan YW.

Genome Res. 2014 Sep;24(9):1526-33. doi: 10.1101/gr.173427.114.

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