Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells

Proceedings of the National Academy of Sciences of the United States of America

Volumn 99, Issue 22, 2002, Pages 14380-14385

  Imren, Suzan ^a   Payen, Emmanuel ^b   Westerman, Karen A ^c,d   Pawliuk, Robert ^c,d   Fabry, Mary E ^e   Eaves, Connie J ^a,f   Cavilla, Benjamin ^a   Wadsworth, Louis D ^g   Beuzard, Yves ^b   Bouhassira, Eric E ^e   Russell, Robert ^e   London, Irving M ^c   Nagel, Ronald L ^e   Leboulch, Philippe ^b,c,d,h   Humphries, R Keith ^a,f  

^a BRITISH COLUMBIA CANCER AGENCY   (Canada)

^b INSERM   (France)

^c MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

^d Genetix Pharmaceuticals   (United States)

^e ALBERT EINSTEIN COLLEGE OF MEDICINE   (United States)

^f UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^g CHILDREN S AND WOMEN S HEALTH CENTRE OF BRITISH COLUMBIA   (Canada)

^h BRIGHAM AND WOMEN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLOBIN; LENTIVIRUS VECTOR;

ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BETA THALASSEMIA; CELL COMPARTMENTALIZATION; CELL COUNT; CELL LINEAGE; CONTROLLED STUDY; ERYTHROCYTE COUNT; GENE DELETION; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSDUCTION; HEMATOPOIETIC CELL; ISOELECTRIC FOCUSING; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; RETICULOCYTE COUNT; STEM CELL TRANSPLANTATION; TREATMENT OUTCOME;

3T3 CELLS; ANIMALS; BETA-THALASSEMIA; CERCOPITHECUS AETHIOPS; COS CELLS; DISEASE MODELS, ANIMAL; ERYTHROCYTES; ERYTHROID PROGENITOR CELLS; GENE EXPRESSION; GENETIC VECTORS; GLOBINS; HUMANS; LENTIVIRUS; LIVER; MICE; MICE, INBRED C57BL; SPLEEN; VIRUS INTEGRATION;

ANIMALIA; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRUS; MURINAE;

EID: 0037195091     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.212507099     Document Type: Article

References (29)

1. Weatherall, D. J. & Clegg, J. B. (1996) Nat. Med. 2, 847-849.
2. Olivieri, N. F. (1999) N. Engl. J. Med. 341, 99-109.
3. Tuan, D. & London, I. M. (1984) Proc. Natl. Acad. Sci. USA 81, 2718-2722.
4. Tuan, D., Solomon, W., Li, Q. & London, I. M. (1985) Proc. Natl. Acad. Sci. USA 82, 6384-6388.
5. Tuan, D. Y., Solomon, W. B., London, I. M. & Lee, D. P. (1989) Proc. Natl. Acad. Sci. USA 86, 2554-2558.
6. Grosveld, F., van Assendelft, G. B., Greaves, D. R. & Kollias, G. (1987) Cell 51, 975-985.
7. Novak, U., Harris, E. A. S., Forrester, W., Groudine, M. & Gelinas, R. (1990) Proc. Natl. Acad. Sci. USA 87, 3386-3390.
8. Chang, J. C., Liu, D. & Kan, Y. W. (1992) Proc. Natl. Acad. Sci. USA 89, 3107-3110.
9. Leboulch, P., Huang, G. M. S., Humphries, R. K., Oh, Y. H., Eaves, C. J., Tuan, D. Y. H. & London, I. M. (1994) EMBO J. 13, 3065-3076.
10. Leboulch, P., Takekoshi, K. J., Pawliuk, R., Humphries, R. K., London, I. M., Eaves, C. J. & Nagel, R. (1995) in Sickle Cell Disease and Thalassaemias: New Trends in Therapy, eds. Beuzard, Y., Lubin, B. & Rosa, J. (John Libbey Eurotext, London), pp. 125-133.
11. Sadelain, M., Wang, C. H., Antoniou, M., Grosveld, F. & Mulligan, R. C. (1995) Proc. Natl. Acad. Sci. USA 92, 6728-6732.
12. Kalberer, C. P., Pawliuk, R., Imren, S., Bachelot, T., Takekoshi, K. J., Fabry, M., Eaves, C. J., London, I. M., Humphries, R. K. & Leboulch, P. (2000) Proc. Natl. Acad. Sci. USA 97, 5411-5415.
13. Raftopoulos, H., Ward, M., Leboulch, P. & Bank, A. (1997) Blood 90, 3414-3422.
14. Naldini, L. & Verma, I. M. (2000) Adv. Virus Res. 55, 599-609.
15. May, C., Rivella, S., Callegari, J., Heller, G., Gaensler, K. M., Luzzatto, L. & Sadelain, M. (2000) Nature 406, 82-86.
16. May, C., Rivella, S., Chadburn, A. & Sadelain, M. (2002) Blood 99, 1902-1908.
17. Pawliuk, R., Westerman, K. A., Fabry, M. E., Payen, E., Tighe, R., Bouhasira, E. E., Acharya, S. A., Ellis, J., London, I. M., Eaves, C. J., et al. (2001) Science 294, 2368-2371.
18. Zennou, V., Petit, C., Guetard, D., Nerhbass, U., Montagnier, L. & Charneau, P. (2000) Cell 101, 173-185.
19. Skow, L. C., Burkhart, B. A., Johnson, F. M., Popp, R. A., Popp, D. M., Goldberg, S. Z., Anderson, W. F., Barnett, L. B. & Lewis, S. E. (1983) Cell 34, 1043-1052.
20. Pawliuk, R., Kay, R., Lansdorp, P. & Humphries, R. K. (1994) Blood 84, 2868-2877.
21. Fabry, M. E., Costantini, F., Pachnis, A., Suzuka, S. M., Bank, N., Aynedjian, H. S., Factor, S. M. & Nagel, R. L. (1992) Proc. Natl. Acad. Sci. USA 89, 12155-12159.
22. Rouyer-Fessard, P., Garel, M. C., Domenget, C., Guetarni, D., Bachir, D., Colonna, P. & Beuzard, Y. (1989) J. Biol. Chem. 264, 19092-19098.
23. Fabry, M. E., Benjamin, L., Lawrence, C. & Nagel, R. L. (1984) Blood 64, 559-563.
24. Bouhassira, E. E., Westerman, K. & Leboulch, P. (1997) Blood 90, 3332-3344.
25. Alami, R., Gilman, J. G., Feng, Y. Q., Marmorato, A., Rochlin, I., Suzuka, S. M., Fabry, M. E., Nagel, R. L. & Bouhassira, E. E. (1999) Blood Cells Mol. Dis. 25, 110-119.
26. Blouin, M. J., Beauchemin, H., Wright, A., De Paepe, M., Sorette, M., Bleau, A. M., Nakamoto, B., Ou, C. N., Stamatoyannopoulos, G. & Trudel, M. (2000) Nat. Med. 6, 177-182.
27. Persons, D. A., Allay, E. R., Sabatino, D. E., Kelly, P., Bodine, D. M. & Nienhuis, A. W. (2001) Blood 97, 3275-3282.
28. Bell, A. C., West, A. G. & Felsenfeld, G. (1999) Cell 98, 387-396.
29. Elowitz, M. B., Levine, A. J., Siggia; E. D. & Swain, P. S. (2002) Science 297, 1183-1186.