Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice

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

Volumn 102, Issue 31, 2005, Pages 11053-11058

  Santat, Leah ^a   Paz, Helicia ^a   Wong, Christie ^a   Li, Lijing ^a   Macer, James ^b   Forman, Stephen ^a   Wong, K K ^a   Chatterjee, Saswati ^a  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

Adeno associated virus; Gene transfer; Hematopoiesis; HIV

Indexed keywords

CD34 ANTIGEN; CD38 ANTIGEN; COMPLEMENTARY RNA; PARVOVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BONE MARROW; CLONOGENESIS; CONTROLLED STUDY; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; IMMUNE DEFICIENCY; IN VIVO STUDY; MACROPHAGE; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROGENY; RECOMBINANT ADENO ASSOCIATED VIRUS 2; SCID MOUSE; SIGNAL TRANSDUCTION; STEM CELL; UMBILICAL CORD BLOOD; VIRUS INHIBITION; VIRUS REPLICATION;

ADP-RIBOSYL CYCLASE; ANIMALS; ANTIGENS, CD; ANTIGENS, CD34; ANTIGENS, CD38; COLONY-FORMING UNITS ASSAY; DEPENDOVIRUS; FETAL BLOOD; GENETIC VECTORS; GRAFT SURVIVAL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HIV LONG TERMINAL REPEAT; HIV-1; HUMANS; MALE; MEMBRANE GLYCOPROTEINS; MICE; MICE, INBRED NOD; MICE, SCID; RNA, ANTISENSE; RNA, VIRAL; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1;

EID: 23344432113     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.0502902102     Document Type: Article

References (35)

1. Emery, D. W., Nishino, T., Murata, K., Fragkos, M. & Stamatoyannopoulos, G. (2002) Int. J. Hematol. 75, 228-236.
2. Kohn, D. B., Sadelain, M., Dunbar, C., Bodine, D., Kiem, H. P., Candotti, F., Tisdale, J., Riviere, I., Blau, C. A., Richard, R. E., et al. (2003) Mol. Ther. 8, 180-187.
3. Cavazzana-Calvo, M., Hacein-Bey, S., de Saint, B. G., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J. L., et al. (2000) Science 288, 669-672.
4. Hacein-Bey-Abina, S., Le Deist, F., Carlier, F., Bouneaud, C., Hue, C., De Villartay, J. P., Thrasher, A. J., Wulffraat, N., Sorensen, R., Dupuis-Girod, S., et al. (2002) N. Engl. J. Med. 346, 1185-1193.
5. Berns, K. I. & Giraud, C. (1996) Curr. Top. Microbiol. Immunol. 218, 1-23.
6. Podsakoff, G., Wong, K. K., Jr., & Chatterjee, S. (1994) J. Virol. 68, 5656-5666.
7. Kessler, P. D., Podsakoff, G. M., Chen, X., McQuiston, S. A., Colosi, P. C., Matelis, L. A., Kurtzman, G. J. & Byrne, B. J. (1996) Proc. Natl. Acad. Sci. USA 93, 14082-14087.
8. Hermonat, P. L. (1994) Cancer Res. 54, 2278-2281.
9. Raj, K., Ogston, P. & Beard, P. (2001) Nature 412, 914-917.
10. Zhou, S. Z., Broxmeyer, H. E., Cooper, S., Harrington, M. A. & Srivastava, A. (1993) Exp. Hematol. 21, 928-933.
11. Einerhand, M. P., Antoniou, M., Zolotukhin, S., Muzyczka, N., Berns, K. I., Grosveld, F. & Valerio, D. (1995) Gene Ther. 2, 336-343.
12. Fisher-Adams, G., Wong, K. K., Jr., Podsakoff, G., Forman, S. J. & Chatterjee, S. (1996) Blood 88, 492-504.
13. Ponnazhagan, S., Yoder, M. C. & Srivastava, A. (1997) J. Virol. 71, 3098-3104.
14. Chatterjee, S., Li, W., Wong, C. A., Fisher-Adams, G., Lu, D., Guha, M., Macer, J. A., Forman, S. J. & Wong, K. K., Jr. (1999) Blood 93, 1882-1894.
15. Srivastava, A. (2004) Methods Mol. Biol. 246, 245-254.
16. Ferrari, F. K., Samulski, T., Shenk, T. & Samulski, R. J. (1996) J. Virol. 70, 3227-3234.
17. Fisher, K. J., Gao, G. P., Weitzman, M. D., DeMatteo, R., Burda, J. F. & Wilson, J. M. (1996) J. Virol. 70, 520-532.
18. Duan, D., Li, Q., Kao, A. W., Yue, Y., Pessin, J. E. & Engelhardt, J. F. (1999) J. Virol. 73, 10371-10376.
19. Xiao, W., Warrington, K. H., Jr., Hearing, P., Hughes, J. & Muzyczka, N. (2002) J. Virol. 76, 11505-11517.
20. Qing, K., Li, W., Zhong, L., Tan, M., Hansen, J., Weigel-Kelley, K. A., Chen, L., Yoder, M. C. & Srivastava, A. (2003) J. Virol. 77, 2741-2746.
21. Thomas, C. E., Storm, T. A., Huang, Z. & Kay, M. A. (2004) J. Virol. 78, 3110-3122.
22. Chatterjee, S., Johnson, P. R. & Wong, K. K., Jr. (1992) Science 258, 1485-1488.
23. Bauer, G., Valdez, P., Kearns, K., Bahner, I., Wen, S. F., Zaia, J. A. & Kohn, D. B. (1997) Blood 89, 2259-2267.
24. Flotte, T. R., Afione, S. A. & Zeitlin, P. L. (1994) Am. J. Respir. Cell Mol. Biol. 11, 517-521.
25. Duan, D., Sharma, P., Yang, J., Yue, Y., Dudus, L., Zhang, Y., Fisher, K. J. & Engelhardt, J. F. (1998) J. Virol. 72, 8568-8577.
26. Kearns, W. G., Afione, S. A., Fulmer, S. B., Pang, M. C., Erikson, D., Egan, M., Landrum, M. J., Flotte, T. R. & Cutting, G. R. (1996) Gene Ther. 3, 748-755.
27. Nakai, H., Montini, E., Fuess, S., Storm, T. A., Grompe, M. & Kay, M. A. (2003) Nat. Genet. 34, 297-302.
28. McCarty, D. M., Young, S. M., Jr., & Samulski, R. J. (2004) Annu. Rev. Genet. 38, 819-845.
29. Song, S., Witek, R. P., Lu, Y., Choi, Y.-K., Zheng, D., Jorgensen, M., Li, C., Flotte, T. R. & Petersen, B. E. (2004) Hepatology 40, 918-924.
30. Chamberlain, J. R., Schwarze, U., Wang, P. R., Hirata, R. K., Hankenson, K. D., Pace, J. M., Underwood, R. A., Song, K. M., Sussman, M., Byers, P. H., et al. (2004) Science 303, 1198-1201.
31. Kumar, S., Mahendra, G., Nagy, T. R. & Ponnazhagan, S. (2004) Hum. Gene Ther. 15, 1197-1206.
32. Zeitlin, P. L., Chu, S., Conrad, C., McVeigh, U., Ferguson, K., Flotte, T. R. & Guggino, W. B. (1995) Gene Ther. 2, 623-631.
33. Wu, P., Ye, Y. & Svendsen, C. N. (2002) Gene Ther. 9, 245-255.
34. Smith-Arica, J. R., Thomson, A. J., Ansell, R., Chiorini, J., Davidson, B. & McWhir, J. (2003) Cloning Stem Cells 5, 51-62.
35. Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X. & Wilson, J. M. (2004) J. Virol. 78, 6381-6388.