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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.


Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease.

Zou J, Mali P, Huang X, Dowey SN, Cheng L.

Blood. 2011 Oct 27;118(17):4599-608. doi: 10.1182/blood-2011-02-335554. Epub 2011 Aug 31.


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. Epub 2013 Oct 23.


Rapid mutation of endogenous zebrafish genes using zinc finger nucleases made by Oligomerized Pool ENgineering (OPEN).

Foley JE, Yeh JR, Maeder ML, Reyon D, Sander JD, Peterson RT, Joung JK.

PLoS One. 2009;4(2):e4348. doi: 10.1371/journal.pone.0004348. Epub 2009 Feb 9.


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. Epub 2013 Oct 28.


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. Epub 2015 Mar 20.


Nuclease-stimulated homologous recombination at the human β-globin gene.

Vannocci T, Kurata H, de la Fuente J, Roberts IA, Porter AC.

J Gene Med. 2014 Jan-Feb;16(1-2):1-10. doi: 10.1002/jgm.2758.


Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation.

Huang X, Wang Y, Yan W, Smith C, Ye Z, Wang J, Gao Y, Mendelsohn L, Cheng L.

Stem Cells. 2015 May;33(5):1470-9. doi: 10.1002/stem.1969.


Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells.

Crane AM, Kramer P, Bui JH, Chung WJ, Li XS, Gonzalez-Garay ML, Hawkins F, Liao W, Mora D, Choi S, Wang J, Sun HC, Paschon DE, Guschin DY, Gregory PD, Kotton DN, Holmes MC, Sorscher EJ, Davis BR.

Stem Cell Reports. 2015 Apr 14;4(4):569-77. doi: 10.1016/j.stemcr.2015.02.005. Epub 2015 Mar 12.


Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs.

Sun N, Zhao H.

Biotechnol Bioeng. 2014 May;111(5):1048-53. doi: 10.1002/bit.25018. Epub 2013 Aug 26.


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.


Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system.

Song B, Fan Y, He W, Zhu D, Niu X, Wang D, Ou Z, Luo M, Sun X.

Stem Cells Dev. 2015 May 1;24(9):1053-65. doi: 10.1089/scd.2014.0347. Epub 2015 Feb 5.


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.


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.


Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs.

Xu P, Tong Y, Liu XZ, Wang TT, Cheng L, Wang BY, Lv X, Huang Y, Liu DP.

Sci Rep. 2015 Jul 9;5:12065. doi: 10.1038/srep12065.


A Comprehensive, Ethnically Diverse Library of Sickle Cell Disease-Specific Induced Pluripotent Stem Cells.

Park S, Gianotti-Sommer A, Molina-Estevez FJ, Vanuytsel K, Skvir N, Leung A, Rozelle SS, Shaikho EM, Weir I, Jiang Z, Luo HY, Chui DHK, Figueiredo MS, Alsultan A, Al-Ali A, Sebastiani P, Steinberg MH, Mostoslavsky G, Murphy GJ.

Stem Cell Reports. 2017 Apr 11;8(4):1076-1085. doi: 10.1016/j.stemcr.2016.12.017. Epub 2017 Jan 19.


Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification.

Maeder ML, Thibodeau-Beganny S, Osiak A, Wright DA, Anthony RM, Eichtinger M, Jiang T, Foley JE, Winfrey RJ, Townsend JA, Unger-Wallace E, Sander JD, Müller-Lerch F, Fu F, Pearlberg J, Göbel C, Dassie JP, Pruett-Miller SM, Porteus MH, Sgroi DC, Iafrate AJ, Dobbs D, McCray PB Jr, Cathomen T, Voytas DF, Joung JK.

Mol Cell. 2008 Jul 25;31(2):294-301. doi: 10.1016/j.molcel.2008.06.016.


Gene correction in patient-specific iPSCs for therapy development and disease modeling.

Jang YY, Ye Z.

Hum Genet. 2016 Sep;135(9):1041-58. doi: 10.1007/s00439-016-1691-5. Epub 2016 Jun 2. Review.


Generation and genetic engineering of human induced pluripotent stem cells using designed zinc finger nucleases.

Ramalingam S, London V, Kandavelou K, Cebotaru L, Guggino W, Civin C, Chandrasegaran S.

Stem Cells Dev. 2013 Feb 15;22(4):595-610. doi: 10.1089/scd.2012.0245. Epub 2012 Oct 19.

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