Gene Targeting in Stem Cells from Individuals with Osteogenesis Imperfecta

Science

Volumn 303, Issue 5661, 2004, Pages 1198-1201

  Chamberlain, Joel R ^a   Schwarze, Ulrike ^a   Wang, Pei Rong ^a   Hirata, Roli K ^a   Hankenson, Kurt D ^c   Pace, James M ^a   Underwood, Robert A ^a   Song, Kit M ^d   Sussman, Michael ^e   Byers, Peter H ^a   Russell, David W ^a,b  

^a UNIVERSITY OF WASHINGTON   (United States)

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

^c UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^d SEATTLE CHILDREN S RESEARCH INSTITUTE   (United States)

^e Shriner's Hospital   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BONE; CARTILAGE; DISEASES; GENES; MUSCLE; MUTAGENESIS; TRANSPLANTATION (SURGICAL);

OSTEOGENESIS; STEM CELLS;

CELLS;

COLLAGEN; COLLAGEN TYPE 1; GENE PRODUCT; KANAMYCIN KINASE; PARVOVIRUS VECTOR;

DISEASE;

ADENO ASSOCIATED VIRUS; ADIPOCYTE; ADULT; ALLELE; ANIMAL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; AUTOTRANSPLANTATION; BONE CELL; BONE DEVELOPMENT; BONE MARROW CELL; CARTILAGE CELL; CELL ADHESION; CELL BASED GENE THERAPY; CELL CULTURE; CELL DIFFERENTIATION; CELL ISOLATION; CELL PROLIFERATION; CELL TYPE; CHEMISTRY; COL1A1 GENE; DOMINANT GENE; GENE; GENE MUTATION; GENE TARGETING; GENE THERAPY; GENE VECTOR; GENETIC MANIPULATION; GENETIC RECOMBINATION; GENETICS; HUMAN; HUMAN CELL; MALE; MESENCHYMAL STEM CELL; MESENCHYME; METABOLISM; MOUSE; MUSCLE CELL; NONHUMAN; OSTEOGENESIS IMPERFECTA; PHYSIOLOGY; POINT MUTATION; PRIORITY JOURNAL; STEM CELL; STEM CELL TRANSPLANTATION;

ALLELES; ANIMALS; BONE MARROW CELLS; CELL DIFFERENTIATION; CELLS, CULTURED; COLLAGEN TYPE I; DEPENDOVIRUS; GENE TARGETING; GENE THERAPY; GENETIC VECTORS; HUMANS; KANAMYCIN KINASE; MALE; MESENCHYMAL STEM CELLS; MICE; OSTEOGENESIS; OSTEOGENESIS IMPERFECTA; POINT MUTATION; RECOMBINATION, GENETIC; STEM CELL TRANSPLANTATION;

ADENO-ASSOCIATED VIRUS; ANIMALIA; PARVOVIRUS;

EID: 10744222737     PISSN: 00368075     EISSN: None     Source Type: Journal    
DOI: 10.1126/science.1088757     Document Type: Article

References (22)

1. P. H. Byers, in The Metabolic and Molecular Basis of Inherited Disease, C. R. Scriver, A. L. Beaudet, W. S. Sly, D. Valle, Eds. (McGraw-Hill, New York, 2000), pp. 5241-5286.
2. M. C. Willing, C. J. Pruchno, M. Atkinson, P. H. Byers, Am. J. Hum. Genet. 51, 508 (1992).
3. J. Korkko et al., Am. J. Hum. Genet. 62, 98 (1998).
4. R. Hirata, J. Chamberlain, R. Dong, D. W. Russell, Nature Biotechnol. 20, 735 (2002).
5. J. Bonadio, K. A. Holbrook, R. E. Gelinas, J. Jacob, P. H. Byers, J. Biol. Chem. 260, 1734 (1985).
6. M. Raghunath, P. Bruckner, B. Steinmann, J. Mol. Biol. 236, 940 (1994).
7. E. M. Horwitz et al., Nature Med. 5, 309 (1999).
8. E. M. Horwitz et al., Proc. Natl. Acad. Sci. U.S.A. 99, 8932 (2002).
9. C. Ploetz, E. I. Zycband, D. E. Birk, J. Struct. Biol. 106, 73 (1991).
10. C. D. Constantinou, M. Pack, S. B. Young, D. J. Prockop, Am. J. Hum. Genet. 47, 670 (1990).
11. M. J. Edwards, R. J. Wenstrup, P. H. Byers, D. H. Cohn, Hum. Mutat. 1, 47 (1992).
12. G. A. Wallis, B. J. Starman, A. B. Zinn, P. H. Byers, Am. J. Hum. Genet. 46, 1034 (1990).
13. R. F. Pereira et al., Proc. Natl. Acad. Sci. U.S.A. 95, 1142 (1998).
14. N. S. Fedarko, M. Moerike, R. Brenner, P. G. Robey, U. Vetter, J. Bone Miner. Res. 7, 921 (1992).
15. N. Inoue, R. K. Hirata, D. W. Russell, J. Virol. 73, 7376 (1999).
16. N. Inoue, R. Dong, R. K. Hirata, D. W. Russell, Mol. Ther. 3, 526 (2001).
17. D. W. Russell, R. K. Hirata, Nature Genet. 18, 325 (1998).
18. A. I. Caplan, J. Orthop. Res. 9, 641 (1991).
19. M. F. Pittenger et al., Science 284, 143 (1999).
20. D. J. Prockop, Science 276, 71 (1997).
21. S. Hacein-Bey-Abina et al., Science 302, 415 (2003).
22. We thank G. Priestley and T. Papayannopoulou for NOD/SCID mice; S. Heimfeld for cadaveric bone marrow cells; C. Miller for EM; D. Chen for mutation analysis; C. Xu, R. Dong, and R. Newton for technical assistance; and T. Kyriakides for helpful discussions. This work was supported by grants from the U.S. National Institutes of Health, the Children's Brittle Bone Foundation, and the George F. Odland Research Fund.