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

Cell Research

Volumn 22, Issue 4, 2012, Pages 637-648

  Wang, Yixuan ^a   Zheng, Chen Guang ^b   Jiang, Yonghua ^a   Zhang, Jiqin ^a   Chen, Jiayu ^a   Yao, Chao ^a   Zhao, Qingguo ^a   Liu, Sheng ^a   Chen, Ke ^b   Du, Juan ^b   Yang, Ze ^c   Gao, Shaorong ^a  

^a NATIONAL INSTITUTE OF BIOLOGICAL SCIENCES   (China)

^b Guangxi Zhuang Autonomous Region   (China)

^c BEIJING HOSPITAL   (China)

Author keywords

iPS; thalassemia

Indexed keywords

BETA GLOBIN; HEMOGLOBIN;

ANIMAL; ARTICLE; BETA THALASSEMIA; BIOSYNTHESIS; CYTOLOGY; DISEASE MODEL; FIBROBLAST; GENE THERAPY; GENETICS; HOMOLOGOUS RECOMBINATION; HUMAN; METABOLISM; MOUSE; MOUSE MUTANT; MUTATION; PLURIPOTENT STEM CELL; PRESCHOOL CHILD; TRANSPLANTATION;

ANIMALS; BETA-GLOBINS; BETA-THALASSEMIA; CHILD, PRESCHOOL; DISEASE MODELS, ANIMAL; FIBROBLASTS; GENE THERAPY; HEMOGLOBINS; HOMOLOGOUS RECOMBINATION; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MICE; MICE, SCID; MUTATION;

IPS; MUS;

EID: 84862776968     PISSN: 10010602     EISSN: 17487838     Source Type: Journal    
DOI: 10.1038/cr.2012.23     Document Type: Article

References (36)

1. Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006; 126:663-676.
2. Okita K, Ichisaka T, Yamanaka S. Generation of germlinecompetent induced pluripotent stem cells. Nature 2007; 448:313-317.
3. Wernig M, Meissner A, Foreman R, et al. In vitro reprogramming of fibroblasts into a pluripotent ES-cell-like state. Nature 2007; 448:318-324.
4. Takahashi K, Tanabe K, Ohnuki M, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007; 131:861-872.
5. Park IH, Zhao R, West JA, et al. Reprogramming of human somatic cells to pluripotency with defined factors. Nature 2008; 451:141-146.
6. Yu J, Vodyanik MA, Smuga-Otto K, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007; 318:1917-1920.
7. Dimos JT, Rodolfa KT, Niakan KK, et al. Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons. Science 2008; 321:1218-1221.
8. Ebert AD, Yu J, Rose FF Jr, et al. Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 2009; 457:277-280.
9. Raya A, Rodriguez-Piza I, Guenechea G, et al. Diseasecorrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature 2009; 460:53-59.
10. Soldner F, Hockemeyer D, Beard C, et al. Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell 2009; 136:964-977.
11. Hanna J, Wernig M, Markoulaki S, et al. Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 2007; 318:1920-1923.
12. Wang Y, Jiang Y, Liu S, Sun X, Gao S. Generation of induced pluripotent stem cells from human beta-thalassemia fibroblast cells. Cell Res 2009; 19:1120-1123.
13. Ye L, Chang JC, Lin C, Sun X, Yu J, Kan YW. Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases. Proc Natl Acad Sci USA 2009; 106:9826-9830.
14. Costa M, Dottori M, Sourris K, et al. A method for genetic modification of human embryonic stem cells using electroporation. Nat Protoc 2007; 2:792-796.
15. Liu P, Jenkins NA, Copeland NG. A highly efficient recombineering- based method for generating conditional knockout mutations. Genome Res 2003; 13:476-484.
16. Vodyanik MA, Slukvin II. Hematoendothelial differentiation of human embryonic stem cells. Curr Protoc Cell Biol 2007; Chapter 23:Unit 23.6.
17. Hockemeyer D, Soldner F, Beard C, et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 2009; 27:851-857.
18. Cui X, Ji D, Fisher DA, Wu Y, Briner DM, Weinstein EJ. Targeted integration in rat and mouse embryos with zinc-finger nucleases. Nat Biotechnol 2011; 29:64-67.
19. Song H, Chung SK, Xu Y. Modeling disease in human ESCs using an efficient BAC-based homologous recombination system. Cell Stem Cell 2010; 6:80-89.
20. Liu GH, Suzuki K, Qu J, et al. Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs. Cell Stem Cell 2011; 8:688-694.
21. Li M, Suzuki K, Qu J, et al. Efficient correction of hemoglobinopathy- causing mutations by homologous recombination in integration-free patient iPSCs. Cell Res 2011; 21:1740-1744.
22. Warren L, Manos PD, Ahfeldt T, et al. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 2010; 7:618-630.
23. Anokye-Danso F, Trivedi CM, Juhr D, et al. Highly efficient miRNA-mediated reprogramming of mouse and human somatic cells to pluripotency. Cell Stem Cell 2010; 8:376-388.
24. Miyoshi N, Ishii H, Nagano H, et al. Reprogramming of mouse and human cells to pluripotency using mature microRNAs. Cell Stem Cell 2011; 8:633-638.
25. Kim D, Kim CH, Moon JI, et al. Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cell 2009; 4:472-476.
26. Lister R, Pelizzola M, Kida YS, et al. Hotspots of aberrant epigenomic reprogramming in human induced pluripotent stem cells. Nature 2011; 471:68-73.
27. Ohi Y, Qin H, Hong C, et al. Incomplete DNA methylation underlies a transcriptional memory of somatic cells in human iPS cells. Nat Cell Biol 2011; 13:541-549.
28. Zhao T, Zhang ZN, Rong Z, Xu Y. Immunogenicity of induced pluripotent stem cells. Nature 2011; 474:212-215.
29. Papapetrou EP, Lee G, Malani N, et al. Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol 2011; 29:73-78.
30. Zhou Q, Brown J, Kanarek A, Rajagopal J, Melton DA. In vivo reprogramming of adult pancreatic exocrine cells to betacells. Nature 2008; 455:627-632.
31. Vierbuchen T, Ostermeier A, Pang ZP, Kokubu Y, Sudhof TC, Wernig M. Direct conversion of fibroblasts to functional neurons by defined factors. Nature 2010; 463:1035-1041.
32. Huang P, He Z, Ji S, et al. Induction of functional hepatocytelike cells from mouse fibroblasts by defined factors. Nature 2011; 475:386-389.
33. Ieda M, Fu JD, Delgado-Olguin P, et al. Direct reprogramming of fibroblasts into functional cardiomyocytes by defined factors. Cell 2010; 142:375-386.
34. Szabo E, Rampalli S, Risueno RM, et al. Direct conversion of human fibroblasts to multilineage blood progenitors. Nature 2010; 468:521-526.
35. Agarwal S, Loh YH, McLoughlin EM, et al. Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients. Nature 2010; 464:292-296.
36. Marion RM, Strati K, Li H, et al. Telomeres acquire embryonic stem cell characteristics in induced pluripotent stem cells. Cell Stem Cell 2009; 4:141-154.