A platform for rapid generation of single and multiplexed reporters in human iPSC lines

Scientific Reports

Volumn 5, Issue , 2015, Pages

  Pei, Ying ^a   Sierra, Guadalupe ^b   Sivapatham, Renuka ^a   Swistowski, Andrzej ^b   Rao, Mahendra S ^a   Zeng, Xianmin ^a,b  

^a BUCK INSTITUTE FOR RESEARCH ON AGING   (United States)

^b XCell Science Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CELL LINE; CELL LINEAGE; CHROMOSOME 13; CHROMOSOME 19; GENE EXPRESSION; GENE LOCUS; GENE ORDER; GENE TARGETING; GENE VECTOR; GENETICS; HUMAN; METABOLISM; PLURIPOTENT STEM CELL; PROCEDURES; REPORTER GENE; REPRODUCIBILITY;

CELL LINE; CELL LINEAGE; CHROMOSOMES, HUMAN, PAIR 13; CHROMOSOMES, HUMAN, PAIR 19; GENE EXPRESSION; GENE KNOCK-IN TECHNIQUES; GENE ORDER; GENE TARGETING; GENES, REPORTER; GENETIC LOCI; GENETIC VECTORS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; REPRODUCIBILITY OF RESULTS;

EID: 84924973780     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep09205     Document Type: Article

References (39)

1. Mali, P. et al. RNA-guided human genome engineering via Cas9. Science 339, 823-826; doi:10.1126/science.1232033 (2013).
2. Boch, J. et al. Breaking the code of DNA binding specificity of TAL-type III effectors. Science 326, 1509-1512; doi:10.1126/science.1178811 (2009).
3. Urnov, F. D. et al. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435, 646-651; doi:10.1038/nature03556 (2005).
4. Moscou, M. J. & Bogdanove, A. J. A simple cipher governs DNA recognition by TAL effectors. Science 326, 1501; doi:10.1126/science.1178817 (2009).
5. Shaltouki, A., Peng, J., Liu, Q., Rao, M. S. & Zeng, X. Efficient generation of astrocytes from human pluripotent stem cells in defined conditions. Stem Cells 31, 941-952; doi:10.1002/stem.1334 (2013).
6. Han, Y. et al. Identification by automated screening of a small molecule that selectively eliminates neural stem cells derived from hESCs but not dopamine neurons. PloS one 4, e7155; doi:10.1371/journal.pone.0007155 (2009).
7. Matsa, E., Burridge, P. W. & Wu, J. C. Human stem cells for modeling heart disease and for drug discovery. Sci. Transl. Med. 6, 239ps236; doi:10.1126/scitranslmed.3008921 (2014).
8. Sinnecker, D. et al. Modeling long-QT syndromes with iPS cells. J Cardiovasc Transl Res. 6, 31-36; doi:10.1007/s12265-012-9416-1 (2013).
9. Laustriat, D., Gide, J. & Peschanski, M. Human pluripotent stem cells in drug discovery and predictive toxicology. Biochem. Soc. Trans. 38, 1051-1057; doi:10.1042/BST0381051 (2010).
10. Sinnecker, D., Laugwitz, K. L. &Moretti, A. Induced pluripotent stem cell-derived cardiomyocytes for drug development and toxicity testing. Pharmacol. Ther. 143, 246-252; doi:10.1016/j.pharmthera.2014.03.004 (2014).
11. Shtrichman, R., Germanguz, I.& Itskovitz-Eldor, J. Induced pluripotent stem cells (iPSCs) derived from different cell sources and their potential for regenerative and personalized medicine. Curr. Mol. Med. 13, 792-805 (2013).
12. Kumar, K. K., Aboud, A. A.&Bowman, A. B. The potential of induced pluripotent stem cells as a translational model for neurotoxicological risk. Neurotoxicology 33, 518-529; doi:10.1016/j.neuro.2012.02.005 (2012).
13. Ananiev, G., Williams, E. C., Li, H. & Chang, Q. Isogenic pairs of wild type and mutant induced pluripotent stem cell (iPSC) lines from Rett syndrome patients as in vitro disease model. PLoS One 6, e25255; doi:10.1371/journal.pone.0025255 (2011).
14. Vojnits, K. & Bremer, S. Challenges of using pluripotent stem cells for safety assessments of substances. Toxicology 270, 10-17; doi:10.1016/j.tox.2009.12.003 (2010).
15. Fu, X. & Xu, Y. Challenges to the clinical application of pluripotent stem cells: towards genomic and functional stability. GenomeMed. 4, 55; doi:10.1186/gm354 (2012).
16. Ho, P. J., Yen, M. L., Yet, S. F. & Yen, B. L. Current applications of human pluripotent stem cells: possibilities and challenges. Cell transplantation 21, 801-814; doi:10.3727/096368911X627507 (2012).
17. Sun, X., Tan, G. & Liew, R. Future challenges for patient-specific induced pluripotent stem cells in cardiovascular medicine. Expert Rev. Cardiovasc. Ther. 10, 943-945; doi:10.1586/erc.12.88 (2012).
18. Tabar, V. & Studer, L. Pluripotent stem cells in regenerative medicine: challenges and recent progress. Nat. Rev. Genet. 15, 82-92; doi:10.1038/nrg3563 (2014).
19. Wang, J. et al. Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme. Genome Res. 22, 1316-1326; doi:10.1101/gr.122879.111 (2012).
20. Holkers, M. et al. Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells. Nucleic Acids Res. 41, e63; doi:10.1093/nar/gks1446 (2013).
21. Luo, Y. et al. Stable Enhanced Green Fluorescent Protein Expression After Differentiation and Transplantation of Reporter Human Induced Pluripotent Stem Cells Generated by AAVS1 Transcription Activator-Like Effector Nucleases. Stem Cells Transl. Med. 3, 821-835; doi:10.5966/sctm.2013-0212 (2014).
22. Maggio, I. et al. Adenoviral vector delivery of RNA-guided CRISPR/Cas9 nuclease complexes induces targeted mutagenesis in a diverse array of human cells. Sci. Rep. 4, 5105; doi:10.1038/srep05105 (2014).
23. Macarthur, C. C. et al. Chromatin insulator elements block transgene silencing in engineered human embryonic stem cell lines at a defined chromosome 13 locus. Stem Cells Dev. 21, 191-205; doi:10.1089/scd.2011.0163 (2012).
24. Yan, Y. et al. Efficient and rapid derivation of primitive neural stem cells and generation of brain subtype neurons from human pluripotent stem cells. Stem Cells Transl. Med. 2, 862-870; doi:10.5966/sctm.2013-0080 (2013).
25. Swistowska, A. M. et al. Stage-specific role for shh in dopaminergic differentiation of human embryonic stem cells induced by stromal cells. Stem Cells Dev. 19, 71-82; doi:10.1089/scd.2009.0107 (2010).
26. Cerbini, T. et al. TALEN-mediated CLYBL targeting enables enhanced transgene expression and one-step generation of multiplexed reporter human iPSC and NSC lines. PloS one In Press (2014).
27. Luo, Y., Rao, M. & Zou, J. Generation of GFP Reporter Human Induced Pluripotent Stem Cells Using AAVS1 Safe Harbor Transcription Activator-Like Effector Nuclease. Curr. Protoc. Stem Cell Biol. 29, 5A 7 1-5A 7 18; doi:10.1002/9780470151808.sc05a07s29 (2014).
28. Tiyaboonchai, A. et al. Utilization of the AAVS1 safe harbor locus for hematopoietic specific transgene expression and gene knockdown in human ES cells. Stem cell Res. 12, 630-637; doi:10.1016/j.scr.2014.02.004 (2014).
29. Owens, R. A. Second generation adeno-associated virus type 2-based gene therapy systems with the potential for preferential integration into AAVS1. Curr. Gene Ther. 2, 145-159 (2002).
30. Recchia, A. & Mavilio, F. Site-specific integration by the adeno-associated virus rep protein. Curr. Gene Ther. 11, 399-405 (2011).
31. Thyagarajan, B. et al. Creation of engineered human embryonic stem cell lines using phiC31 integrase. Stem cells 26, 119-126; doi:10.1634/stemcells.2007-0283 (2008).
32. Liu, Y. et al. Generation of platform human embryonic stem cell lines that allow efficient targeting at a predetermined genomic location. Stem Cells Dev. 18, 1459-1472; doi:10.1089/scd.2009.0047 (2009).
33. Sadelain, M., Papapetrou, E. P. &Bushman, F. D. Safe harbours for the integration of new DNA in the human genome. Nat. Rev. Cancer 12, 51-58; doi:10.1038/nrc3179 (2012).
34. Zhu, H., Lensch, M. W., Cahan, P. & Daley, G. Q. Investigating monogenic and complex diseases with pluripotent stem cells. Nat. Rev. Genet. 12, 266-275; doi:10.1038/nrg2951 (2011).
35. Lie, K. H., Chung, H. C. & Sidhu, K. S. Derivation, propagation, and characterization of neuroprogenitors from pluripotent stem cells (hESCs and hiPSCs). Methods Mol. Biol. 873, 237-246; doi:10.1007/978-1-61779-794-1-15 (2012).
36. Zou, J. et al. Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood 117, 5561-5572; doi:10.1182/blood-2010-12-328161 (2011).
37. Swistowski, A. et al. Xeno-free defined conditions for culture of human embryonic stem cells, neural stem cells and dopaminergic neurons derived from them. PloS one 4, e6233; doi:10.1371/journal.pone.0006233 (2009).
38. Zeng, X. et al. Stable expression of hrGFP by mouse embryonic stem cells: promoter activity in the undifferentiated state and during dopaminergic neural differentiation. Stem Cells 21, 647-653; doi:10.1634/stemcells.21-6-647 (2003).
39. Liu, Q. et al.Human neural crest stem cells derived from human ESCs and induced pluripotent stem cells: induction, maintenance, and differentiation into functional schwann cells. Stem Cells Transl. Med. 1, 266-278; doi:10.5966/sctm.2011-0042 (2012).