Reprogramming based gene therapy for inherited red blood cell disorders

Cell Research

Volumn 22, Issue 6, 2012, Pages 941-944

  Xu, Xiuling ^a   Qu, Jing ^a,b   Suzuki, Keiichiro ^b   Li, Mo ^b   Zhang, Weizhou ^d   Liu, Guang Hui ^a   Belmonte, Juan Carlos Izpisua ^b,c  

^a INSTITUTE OF BIOPHYSICS   (China)

^b SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^c CENTER OF REGENERATIVE MEDICINE IN BARCELONA   (Spain)

^d San Diego   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLOBIN;

ARTICLE; CYTOLOGY; GENE THERAPY; GENETICS; HEMATOLOGIC DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; METABOLISM; NUCLEAR REPROGRAMMING; PATHOLOGY; PLURIPOTENT STEM CELL; SICKLE CELL ANEMIA; THALASSEMIA;

ANEMIA, SICKLE CELL; BETA-GLOBINS; GENE THERAPY; HEMATOLOGIC DISEASES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INDUCED PLURIPOTENT STEM CELLS; NUCLEAR REPROGRAMMING; THALASSEMIA;

EID: 84861865179     PISSN: 10010602     EISSN: 17487838     Source Type: Journal    
DOI: 10.1038/cr.2012.54     Document Type: Article

References (18)

1. Loustau V, Guillaud C, Garcon L, Godeau B, Michel M. Hemolytic anemia in adults: Main causes and diagnostic procedure. Presse Med 2011; 40:470-485.
2. Douay L. In vitro generation of red blood cells for transfusion: a model for regenerative medicine. Regen Med 2012; 7:1-2.
3. 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.
4. 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.
5. Wang Y, Zheng CG, Jiang Y, et al. Genetic correction of beta-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice. Cell Res 2012; 22:637-648.
6. Zou J, Mali P, Huang X, Dowey SN, Cheng L. Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. Blood 2011; 118:4599-4608.
7. Mali P, Chou BK, Yen J, et al. Butyrate greatly enhances derivation of human induced pluripotent stem cells by promoting epigenetic remodeling and the expression of pluripotency-associated genes. Stem Cells 2010; 28:713-720.
8. Sebastiano V, Maeder ML, Angstman JF, et al. In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases. Stem Cells 2011; 29:1717-1726.
9. 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.
10. Liu GH, Suzuki K, Qu J, et al. Targeted gene correction of laminopathyassociated LMNA mutations in patientspecific iPSCs. Cell Stem Cell 2011; 8:688-694.
11. Raya A, Rodriguez-Piza I, Guenechea G, et al. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature 2009; 460:53-59.
12. Raya A, Rodriguez-Piza I, Navarro S, et al. A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells. Nat Protoc 2010; 5:647-660.
13. Muller LU, Milsom MD, Harris CE, et al. Overcoming reprogramming resistance of fanconi anemia cells. Blood 2012 Feb 27. doi:10.1182/blood-2012- 02-408674
14. 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.
15. Chang KH, Nelson AM, Cao H, et al. Definitive-like erythroid cells derived from human embryonic stem cells coexpress high levels of embryonic and fetal globins with little or no adult globin. Blood 2006; 108:1515-1523.
16. Qiu C, Olivier EN, Velho M, Bouhassira EE. Globin switches in yolk saclike primitive and fetal-like definitive red blood cells produced from human embryonic stem cells. Blood 2008; 111:2400-2408.
17. Chang KH, Huang A, Hirata RK, Wang PR, Russell DW, Papayannopoulou T. Globin phenotype of erythroid cells derived from human induced pluripotent stem cells. Blood 2010; 115:2553-2554.
18. Szabo E, Rampalli S, Risueno RM, et al. Direct conversion of human fibroblasts to multilineage blood progenitors. Nature 2010; 468:521-526.