Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

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

Volumn 97, Issue 25, 2000, Pages 13714-13719

  Wang, Bing ^a   Li, Juan ^a   Xiao, Xiao ^a,b  

^a UNIVERSITY OF PITTSBURGH   (United States)

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

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; DYSTROPHIN; GENE PRODUCT; VIRUS VECTOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL MEMBRANE; CELL TRANSPLANTATION; COMPLEX FORMATION; CONTROLLED STUDY; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; GENE EXPRESSION; GENE MUTATION; GENE THERAPY; GENE TRANSFER; HUMAN; MOLECULAR STABILITY; MOUSE; MUSCLE CELL; MYOBLAST; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; TRANSGENIC MOUSE; TREATMENT OUTCOME;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DYSTROPHIN; GENETIC VECTORS; HUMANS; MICE; MICE, INBRED MDX; MUSCULAR DYSTROPHY, DUCHENNE;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA; MUS MUSCULUS;

EID: 0034610364     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.240335297     Document Type: Article

References (39)

1. Kunkel, L. M. (1986) Nature (London) 322, 73-77.
2. Koenig, M., Hoffman, E. P., Bertelson, C. J., Monaco, A. P., Feener, C. & Kunkel, L. M. (1987) Cell 50, 509-517.
3. Hoffman, E. P., Brown, R. H., Jr. & Kunkel, L. M. (1987) Cell 51, 919-928.
4. Koenig, M. & Kunkel, L. M. (1990) J. Biol. Chem. 265, 4560-4566.
5. Gussoni, E., Blau, H. M. & Kunkel, L. M. (1997) Nat. Med. 3, 970-977.
6. Acsadi, G., Dickson, G., Love, D. R., Jani, A., Walsh, F. S., Gurusinghe, A., Wolff, J. A. & Davies, K. E. (1991) Nature (London) 352, 815-818.
7. Rando, T. A., Disatnik, M. H. & Zhou, L. Z. (2000) Proc. Natl. Acad. Sci. USA 97, 5363-5368.
8. Ragot, T., Vincent, N., Chafey, P., Vigne, E., Gilgenkrantz, H., Couton, D., Cartaud, J., Briand, P., Kaplan, J. C., Perricaudet, M., et al. (1993) Nature (London) 361, 647-650.
9. Howell, J. M., Lochmuller, H., O'Hara, A., Fletcher, S., Kakulas, B. A., Massie, B., Nalbantoglu, J. & Karpati, G. (1998) Hum. Gene Ther. 9, 629-634.
10. Muzyczka, N. (1992) Curr. Top. Microbiol. Immunol. 158, 97-129.
11. Kaplitt, M. G., Leone, P., Samulski, R. J., Xiao, X., Pfaff, D. W., O'Malley, K. L. & During, M. J. (1994) Nat. Genet. 8, 148-154.
12. Xiao, X., Li, J. & Samulski, R. J. (1996) J. Virol. 70, 8098-8108.
13. 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.
14. Xiao, W., Berta, S. C., Lu, M. M., Moscioni, A. D., Tazelaar, J. & Wilson, J. M. (1998) J. Virol. 72, 10222-10226.
15. Pruchnic, R., Cao, B., Peterson, Z. Q., Xiao, X., Li, J., Samulski, R. J., Epperly, M. & Huard, J. (2000) Hum. Gene Ther. 11, 521-536.
16. Greelish, J. P., Su, L. T., Lankford, E. B., Burkman, J. M., Chen, H., Konig, S. K., Mercier, I. M., Desjardins, P. R., Mitchell, M. A., Zheng, X. G., et al. (1999) Nat. Med. 5, 439-443.
17. Xiao, X., Li, J., Tsao, T.-Y., Dressman, D., Hoffman, E. P. & Watchko, J. F. (2000) J. Virol. 74, 1436-1442.
18. Cordier, L., Hack, A. A., Scott, M. O., Barton-Davis, E. R., Gao, G.-P., Wilson, J. M., McNally, E. M. & Sweeney, H. L. (2000) Mol. Ther. 1, 119-129.
19. Sun, L., Li, J. & Xiao, X. (2000) Nat. Med. 6, 599-602.
20. Duan, D., Yue, Y., Yan, Z. & Engelhardt, J. F. (2000) Nat. Med. 6, 595-598.
21. Song, S., Morgan, M., Ellis, T., Poirier, A., Chesnut, K., Wang, J., Brantly, M., Muzyczka, N., Byrne, B. J., Atkinson, M. & Flotte, T. R. (1998) Proc. Natl. Acad. Sci. USA 95, 14384-14388.
22. Kay, M. A., Manno, C. S., Ragni, M. V., Larson, P. J., Couto, L. B., McClelland, A., Glader, B., Chew, A. J., Tsai, S. J., Herzog, R. W., et al. (2000) Nat. Genet. 24, 257-261.
23. Shield, M. A., Haugen, H. S., Clegg, C. H. & Hauschka, S. D. (1996) Mol. Cell. Biol. 16, 5058-5068.
24. Flotte, T. R., Afione, S. A., Solow, R., Drumm, M. L., Markakis, D., Guggino, W. B., Zeitlin, P. L. & Carter, B. J. (1993) J. Biol. Chem. 268, 3781-3790.
25. Xiao, X., Li, J. & Samulski, R. J. (1998) J. Virol. 72, 2224-2232.
26. Snyder, R., Xiao, X. & Samulski, R. J. (1996) in Current Protocols in Human Genetics, eds. Dracopoli, N., Haines, J., Krof, B., Moir, D., Seidman, C., Seidman, J. & Smith, D. (Wiley, New York), pp. 12.1.1-12.2.23.
27. Li, J., Dressman, D., Tsao, Y. P., Sakamoto, A., Hoffman, E. P. & Xiao, X. (1999) Gene Ther. 6, 74-82.
28. Matsuda, R., Nishikawa, A. & Tanaka, H. (1995) J. Biochem. (Tokyo) 118, 959-964.
29. England, S. B., Nicholson, L. V., Johnson, M. A., Forrest, S. M., Love, D. R., Zubrzycka-Gaarn, E. E., Bulman, D. E., Harris, J. B. & Davies, K. E. (1990) Nature (London) 343, 180-182.
30. Passos-Bueno, M. R., Vainzof, M., Marie, S. K. & Zatz, M. (1994) Hum. Mol. Genet. 3, 919-922.
31. Mirabella, M., Galluzzi, G., Manfredi, G., Bertini, E., Ricci, E., De Leo, R., Tonali, P. & Servidei, S. (1998) Neurology 51, 592-595.
32. Rafael, J. A., Cox, G. A., Corrado, K., Jung, D., Campbell, K. P. & Chamberlain, J. S. (1996) J. Cell Biol. 134, 93-102.
33. Corrado, K., Rafael, J. A., Mills, P. L., Cole, N. M., Faulkner, J. A., Wang, K. & Chamberlain, J. S. (1996) J. Cell Biol. 134, 873-884.
34. Fanin, M., Freda, M. P., Vitiello, L., Danieli, G. A., Pegoraro, E. & Angelini, C. (1996) Muscle Nerve 19, 1154-1160.
35. Martin, H. & Ontell, M. (1988) Muscle Nerve 11, 588-596.
36. Yuasa, K., Miyagoe, Y., Yamamoto, K., Nabeshima, Y., Dickson, G. & Takeda, S. (1998) FEBS Lett. 425, 329-336.
37. Yamamoto, K., Yuasa, K., Miyagoe, Y., Hosaka, Y., Tsukita, K., Yamamoto, H., Nabeshima, Y. I. & Takeda, S. (2000) Hum. Gene Ther. 11, 669-680.
38. Cox, G. A., Sunada, Y., Campbell, K. P. & Chamberlain, J. S. (1994) Nat. Genet. 8, 333-339.
39. Gussoni, E., Soneoka, Y., Strickland, C. D., Buzney, E. A., Khan, M. K., Flint, A. F., Kunkel, L. M. & Mulligan, R. C. (1999) Nature (London) 401, 390-394.