Disease-Specific Induced Pluripotent Stem Cells

Cell

Volumn 134, Issue 5, 2008, Pages 877-886

  Park, In Hyun ^a,g   Arora, Natasha ^a,g   Huo, Hongguang ^a,g   Maherali, Nimet ^b,c,g   Ahfeldt, Tim ^b,e,g   Shimamura, Akiko ^d   Lensch, M William ^a,g   Cowan, Chad ^b,f,g   Hochedlinger, Konrad ^b,g   Daley, George Q ^a,g,h  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL CANCER CENTER   (United States)

^c HARVARD UNIVERSITY   (United States)

^d UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^e UNIVERSITY MEDICAL CENTER HAMBURG EPPENDORF   (Germany)

^f STOWERS INSTITUTE FOR MEDICAL RESEARCH   (United States)

^g HARVARD STEM CELL INSTITUTE   (United States)

^h BRIGHAM AND WOMEN S HOSPITAL   (United States)

Author keywords

HUMDISEASE; STEMCELL

Indexed keywords

ADENOSINE DEAMINASE DEFICIENCY; ADULT; ANIMAL CELL; ARTICLE; BECKER MUSCULAR DYSTROPHY; CELL IMMORTALIZATION; CELL LINE; CELL SPECIFICITY; CELL STIMULATION; CLINICAL ARTICLE; CONTROLLED STUDY; DOWN SYNDROME; DUCHENNE MUSCULAR DYSTROPHY; FEMALE; GAUCHER DISEASE; GENETIC DISORDER; HUMAN; HUMAN CELL; HUNTINGTON CHOREA; IN VITRO STUDY; INHERITANCE; INSULIN DEPENDENT DIABETES MELLITUS; JUVENILE DIABETES MELLITUS; LESCH NYHAN SYNDROME; MALE; MOUSE; MUTATIONAL ANALYSIS; NONHUMAN; PARKINSON DISEASE; PLURIPOTENT STEM CELL; PRESCHOOL CHILD; PRIORITY JOURNAL; SEVERE COMBINED IMMUNODEFICIENCY; SHWACHMAN SYNDROME; STEM CELL EXPANSION; TISSUE CULTURE; TRISOMY 21;

IPS;

EID: 50549089957     PISSN: 00928674     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.cell.2008.07.041     Document Type: Article

References (32)

1. Antonarakis S.E., Lyle R., Dermitzakis E.T., Reymond A., and Deutsch S. Chromosome 21 and down syndrome: from genomics to pathophysiology. Nat. Rev. Genet. 5 (2004) 725-738
2. Austin K.M., Leary R.J., and Shimamura A. The Shwachman-Diamond SBDS protein localizes to the nucleolus. Blood 106 (2005) 1253-1258
3. Beggs A.H., Koenig M., Boyce F.M., and Kunkel L.M. Detection of 98% of DMD/BMD gene deletions by polymerase chain reaction. Hum. Genet. 86 (1990) 45-48
4. Beutler E., Gelbart T., Kuhl W., Sorge J., and West C. Identification of the second common Jewish Gaucher disease mutation makes possible population-based screening for the heterozygous state. Proc. Natl. Acad. Sci. USA 88 (1991) 10544-10547
5. Bittles A.H., Bower C., Hussain R., and Glasson E.J. The four ages of Down syndrome. Eur. J. Public Health 17 (2007) 221-225
6. Brambrink T., Foreman R., Welstead G.G., Lengner C.J., Wernig M., Suh H., and Jaenisch R. Sequential expression of pluripotency markers during direct reprogramming of mouse somatic cells. Cell Stem Cell 2 (2008) 151-159
7. Calado R.T., Graf S.A., Wilkerson K.L., Kajigaya S., Ancliff P.J., Dror Y., Chanock S.J., Lansdorp P.M., and Young N.S. Mutations in the SBDS gene in acquired aplastic anemia. Blood 110 (2007) 1141-1146
8. Chamberlain J.S., Gibbs R.A., Ranier J.E., Nguyen P.N., and Caskey C.T. Deletion screening of the Duchenne muscular dystrophy locus via multiplex DNA amplification. Nucleic Acids Res. 16 (1988) 11141-11156
9. Chen M., Tomkins D.J., Auerbach W., McKerlie C., Youssoufian H., Liu L., Gan O., Carreau M., Auerbach A., Groves T., et al. Inactivation of Fac in mice produces inducible chromosomal instability and reduced fertility reminiscent of Fanconi anaemia. Nat. Genet. 12 (1996) 448-451
10. Chong S.S., Almqvist E., Telenius H., LaTray L., Nichol K., Bourdelat-Parks B., Goldberg Y.P., Haddad B.R., Richards F., Sillence D., et al. Contribution of DNA sequence and CAG size to mutation frequencies of intermediate alleles for Huntington disease: evidence from single sperm analyses. Hum. Mol. Genet. 6 (1997) 301-309
11. Di Giorgio F.P., Carrasco M.A., Siao M.C., Maniatis T., and Eggan K. Non-cell autonomous effect of glia on motor neurons in an embryonic stem cell-based ALS model. Nat. Neurosci. 10 (2007) 608-614
12. Doetschman T., Gregg R.G., Maeda N., Hooper M.L., Melton D.W., Thompson S., and Smithies O. Targeted correction of a mutant HPRT gene in mouse embryonic stem cells. Nature 330 (1987) 576-578
13. Doetschman T., Maeda N., and Smithies O. Targeted mutation of the Hprt gene in mouse embryonic stem cells. Proc. Natl. Acad. Sci. USA 85 (1988) 8583-8587
14. Grimm S. The art and design of genetic screens: mammalian culture cells. Nat. Rev. Genet. 5 (2004) 179-189
15. Hirschhorn R., Chen A.S., Israni A., Yang D.R., and Huie M.L. Two new mutations at the adenosine deaminase (ADA) locus (Q254X and del nt1050-54) unusual for not being missense mutations. Hum. Mutat. 2 (1993) 320-323
16. Lensch M.W., Schlaeger T.M., Zon L.I., and Daley G.Q. Teratoma formation assays with human embryonic stem cells: a rationale for one type of human-animal chimera. Cell Stem Cell 1 (2007) 253-258
17. Lerou P.H., Yabuuchi A., Huo H., Takeuchi A., Shea J., Cimini T., Ince T.A., Ginsburg E., Racowsky C., and Daley G.Q. Human embryonic stem cell derivation from poor-quality embryos. Nat. Biotechnol. 26 (2008) 212-214
18. Lowry W.E., Richter L., Yachechko R., Pyle A.D., Tchieu J., Sridharan R., Clark A.T., and Plath K. Generation of human induced pluripotent stem cells from dermal fibroblasts. Proc. Natl. Acad. Sci. USA 105 (2008) 2883-2888
19. Murry C.E., and Keller G. Differentiation of embryonic stem cells to clinically relevant populations: lessons from embryonic development. Cell 132 (2008) 661-680
20. Nelson D.L., and Gibbs R.A. Genetics. The critical region in trisomy 21. Science 306 (2004) 619-621
21. Nussbaum R.L., Crowder W.E., Nyhan W.L., and Caskey C.T. A three-allele restriction-fragment-length polymorphism at the hypoxanthine phosphoribosyltransferase locus in man. Proc. Natl. Acad. Sci. USA 80 (1983) 4035-4039
22. Olson L.E., Richtsmeier J.T., Leszl J., and Reeves R.H. A chromosome 21 critical region does not cause specific Down syndrome phenotypes. Science 306 (2004) 687-690
23. Park I.H., Lerou P.H., Zhao R., Huo H., and Daley G.Q. Generation of human-induced pluripotent stem cells. Nat. Protocols 3 (2008) 1180-1186
24. Park I.H., Zhao R., West J.A., Yabuuchi A., Huo H., Ince T.A., Lerou P.H., Lensch M.W., and Daley G.Q. Reprogramming of human somatic cells to pluripotency with defined factors. Nature 451 (2008) 141-146
25. Prior T.W., and Bridgeman S.J. Experience and strategy for the molecular testing of Duchenne muscular dystrophy. J. Mol. Diagn. 7 (2005) 317-326
26. Rideout III W.M., Hochedlinger K., Kyba M., Daley G.Q., and Jaenisch R. Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy. Cell 109 (2002) 17-27
27. Riess O., Noerremoelle A., Soerensen S.A., and Epplen J.T. Improved PCR conditions for the stretch of (CAG)n repeats causing Huntington's disease. Hum. Mol. Genet. 2 (1993) 637
28. Stadtfeld M., Maherali N., Breault D.T., and Hochedlinger K. Defining molecular cornerstones during fibroblast to iPS cell reprogramming in mouse. Cell Stem Cell 2 (2008) 230-240
29. Takahashi K., Tanabe K., Ohnuki M., Narita M., Ichisaka T., Tomoda K., and Yamanaka S. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131 (2007) 861-872
30. Thomas K.R., and Capecchi M.R. Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells. Cell 51 (1987) 503-512
31. Verlinsky Y., Strelchenko N., Kukharenko V., Rechitsky S., Verlinsky O., Galat V., and Kuliev A. Human embryonic stem cell lines with genetic disorders. Reprod. Biomed. Online 10 (2005) 105-110
32. Yu J., Vodyanik M.A., Smuga-Otto K., Antosiewicz-Bourget J., Frane J.L., Tian S., Nie J., Jonsdottir G.A., Ruotti V., Stewart R., et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 318 (2007) 1917-1920