Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs

Cell Reports

Volumn 12, Issue 9, 2015, Pages 1385-1390

  Firth, Amy L ^a   Menon, Tushar ^a   Parker, Gregory S ^a   Qualls, Susan J ^a   Lewis, Benjamin M ^a   Ke, Eugene ^a   Dargitz, Carl T ^a   Wright, Rebecca ^a   Khanna, Ajai ^b   Gage, Fred H ^a   Verma, Inder M ^a  

^a SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^b CLEVELAND CLINIC LERNER COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR;

ARTICLE; CELL DIFFERENTIATION; CELL MATURATION; CELL MEMBRANE; CFTR GENE; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CONTROLLED STUDY; CYSTIC FIBROSIS; GENE DELETION; GENE EXPRESSION; GENE FUNCTION; GENE LOCUS; GENE MUTATION; GENE SEQUENCE; GENE THERAPY; GENETIC DISORDER; GENETIC ENGINEERING; HOMOZYGOTE; HUMAN; HUMAN CELL; HUMAN TISSUE; LUNG ALVEOLUS EPITHELIUM CELL; LUNG FIBROSIS; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; CELL CULTURE; CYTOLOGY; EPITHELIUM CELL; GENETICS; INDUCED PLURIPOTENT STEM CELL; LUNG; METABOLISM; MUTATION; PROCEDURES; TARGETED GENE REPAIR;

CELL DIFFERENTIATION; CELLS, CULTURED; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; EPITHELIAL CELLS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; LUNG; MUTATION; TARGETED GENE REPAIR;

EID: 84940793000     PISSN: None     EISSN: 22111247     Source Type: Journal    
DOI: 10.1016/j.celrep.2015.07.062     Document Type: Article

References (24)

1. Boyd A.C., Porteous D.J. Revisiting the mouse lung model for CF. Gene Ther. 2004, 11:737-738.
2. Cho S.W., Kim S., Kim J.M., Kim J.S. Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat. Biotechnol. 2013, 31:230-232.
3. Cong L., Ran F.A., Cox D., Lin S., Barretto R., Habib N., Hsu P.D., Wu X., Jiang W., Marraffini L.A., Zhang F. Multiplex genome engineering using CRISPR/Cas systems. Science 2013, 339:819-823.
4. Crane A.M., Kramer P., Bui J.H., Chung W.J., Li X.S., Gonzalez-Garay M.L., Hawkins F., Liao W., Mora D., Choi S., et al. Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells. Stem Cell Reports 2015, 4:569-577.
5. DiCarlo J.E., Norville J.E., Mali P., Rios X., Aach J., Church G.M. Genome engineering in Saccharomyces cerevisiae using CRISPR-Cas systems. Nucleic Acids Res. 2013, 41:4336-4343.
6. Firth A.L., Dargitz C.T., Qualls S.J., Menon T., Wright R., Singer O., Gage F.H., Khanna A., Verma I.M. Generation of multiciliated cells in functional airway epithelia from human induced pluripotent stem cells. Proc. Natl. Acad. Sci. USA 2014, 111:E1723-E1730.
7. Fusaki N., Ban H., Nishiyama A., Saeki K., Hasegawa M. Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome. Proc. Jpn. Acad., Ser. B, Phys. Biol. Sci. 2009, 85:348-362.
8. Hale C.R., Majumdar S., Elmore J., Pfister N., Compton M., Olson S., Resch A.M., Glover C.V., Graveley B.R., Terns R.M., Terns M.P. Essential features and rational design of CRISPR RNAs that function with the Cas RAMP module complex to cleave RNAs. Mol. Cell 2012, 45:292-302.
9. Hwang W.Y., Fu Y., Reyon D., Maeder M.L., Tsai S.Q., Sander J.D., Peterson R.T., Yeh J.R., Joung J.K. Efficient genome editing in zebrafish using a CRISPR-Cas system. Nat. Biotechnol. 2013, 31:227-229.
10. Jiang W., Bikard D., Cox D., Zhang F., Marraffini L.A. RNA-guided editing of bacterial genomes using CRISPR-Cas systems. Nat. Biotechnol. 2013, 31:233-239.
11. Jinek M., Chylinski K., Fonfara I., Hauer M., Doudna J.A., Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 2012, 337:816-821.
12. Mali P., Yang L., Esvelt K.M., Aach J., Guell M., DiCarlo J.E., Norville J.E., Church G.M. RNA-guided human genome engineering via Cas9. Science 2013, 339:823-826.
13. Millen A.M., Horvath P., Boyaval P., Romero D.A. Mobile CRISPR/Cas-mediated bacteriophage resistance in Lactococcus lactis. PLoS ONE 2012, 7:e51663.
14. Nakayama T., Fish M.B., Fisher M., Oomen-Hajagos J., Thomsen G.H., Grainger R.M. Simple and efficient CRISPR/Cas9-mediated targeted mutagenesis in Xenopus tropicalis. Genesis 2013, 51:835-843.
15. Patrick A.E., Karamyshev A.L., Millen L., Thomas P.J. Alteration of CFTR transmembrane span integration by disease-causing mutations. Mol. Biol. Cell 2011, 22:4461-4471.
16. Rogan M.P., Stoltz D.A., Hornick D.B. Cystic fibrosis transmembrane conductance regulator intracellular processing, trafficking, and opportunities for mutation-specific treatment. Chest 2011, 139:1480-1490.
17. Rogers C.S., Stoltz D.A., Meyerholz D.K., Ostedgaard L.S., Rokhlina T., Taft P.J., Rogan M.P., Pezzulo A.A., Karp P.H., Itani O.A., et al. Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs. Science 2008, 321:1837-1841.
18. Schwank G., Koo B.K., Sasselli V., Dekkers J.F., Heo I., Demircan T., Sasaki N., Boymans S., Cuppen E., van der Ent C.K., et al. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. Cell Stem Cell 2013, 13:653-658.
19. Sun X., Sui H., Fisher J.T., Yan Z., Liu X., Cho H.J., Joo N.S., Zhang Y., Zhou W., Yi Y., et al. Disease phenotype of a ferret CFTR-knockout model of cystic fibrosis. J. Clin. Invest. 2010, 120:3149-3160.
20. Takahashi K., Okita K., Nakagawa M., Yamanaka S. Induction of pluripotent stem cells from fibroblast cultures. Nat. Protoc. 2007, 2:3081-3089.
21. Takahashi K., Tanabe K., Ohnuki M., Narita M., Ichisaka T., Tomoda K., Yamanaka S. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007, 131:861-872.
22. Wang H., Yang H., Shivalila C.S., Dawlaty M.M., Cheng A.W., Zhang F., Jaenisch R. One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell 2013, 153:910-918.
23. Welsh M.J., Rogers C.S., Stoltz D.A., Meyerholz D.K., Prather R.S. Development of a porcine model of cystic fibrosis. Trans. Am. Clin. Climatol. Assoc. 2009, 120:149-162.
24. Wiedenheft B., Sternberg S.H., Doudna J.A. RNA-guided genetic silencing systems in bacteria and archaea. Nature 2012, 482:331-338.