Cut and paste: Restoring cellular function by gene correction

Cell Research

Volumn 22, Issue 2, 2012, Pages 283-284

  Liu, Guang Hui ^a,b   Sancho Martinez, Ignacio ^a   Belmonte, Juan Carlos Izpisua ^a,c  

^a SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^b INSTITUTE OF BIOPHYSICS   (China)

^c CENTER OF REGENERATIVE MEDICINE IN BARCELONA   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

DEOXYRIBONUCLEASE;

ANIMAL; ARTICLE; BACTERIAL ARTIFICIAL CHROMOSOME; CYTOLOGY; GENE TARGETING; GENETICS; HUMAN; METABOLISM; MOUSE; PLURIPOTENT STEM CELL; REGENERATIVE MEDICINE;

ANIMALS; CHROMOSOMES, ARTIFICIAL, BACTERIAL; DEOXYRIBONUCLEASES; GENE TARGETING; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MICE; REGENERATIVE MEDICINE;

EID: 84856599259     PISSN: 10010602     EISSN: 17487838     Source Type: Journal    
DOI: 10.1038/cr.2011.192     Document Type: Article

References (14)

1. 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.
2. Xu D, Alipio Z, Fink LM, et al. Phenotypic correction of murine hemophilia A using an iPS cell-based therapy. Proc Natl Acad Sci USA 2009; 106:808-813.
3. Li H, Haurigot V, Doyon Y, et al. In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 2011; 475:217-221.
4. Liu GH et al. Recapitulation of premature ageing with iPSCs from Hutchinson- Gilford progeria syndrome. Nature 2011; 472:221-225.
5. 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.
6. Soldner F, Laganière J, Cheng AW, et al. Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell 2011; 146:318-331.
7. Howden SE, Gore A, Li Z, et al. Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy. Proc Natl Acad Sci USA 2011; 108:6537-6542.
8. Papapetrou EP, Lee G, Malani N, et al. Genomic safe harbors permit high ?-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol 2011; 29:73-78.
9. Raya A, Rodríguez-Pizà I, Guenechea G, et al. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature 2009; 460:53-59.
10. Panopoulos AD, Ruiz S, Izpisua Belmonte JC. iPSCs: induced back to controversy. Cell Stem Cell 2011; 8:347-348.
11. Urbach A, Bar-Nur O, Daley GQ, Benvenisty N. Differential modeling of fragile X syndrome by human embryonic stem cells and induced pluripotent stem cells. Cell Stem Cell 2010; 6:407-411.
12. Hockemeyer D, Wang H, Kiani S, et al. Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol 2011; 29:731-734.
13. Yusa K, Rashid ST, Strick-Marchand H, et al. Targeted gene correction of ?1-antitrypsin deficiency in induced pluripotent stem cells. Nature 2011; 478:391-394.
14. 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.