Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotides

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

Volumn 93, Issue 23, 1996, Pages 12840-12844

  Sierakowska, Halina ^a,c   Sambade, Maria J ^a   Agrawal, Sudhir ^b   Kole, Ryszard ^a,d  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^b HYBRIDON INC   (United States)

^c INSTITUTE OF BIOCHEMISTRY AND BIOPHYSICS   (Poland)

^d UNIVERSITY OF NORTH CAROLINA   (United States)

Author keywords

gene therapy; RNA splicing

Indexed keywords

BETA GLOBIN; DNA BINDING PROTEIN; MESSENGER RNA;

ANIMAL CELL; BETA THALASSEMIA; CONFERENCE PAPER; GENE MUTATION; GENE THERAPY; HUMAN; HUMAN CELL; NONHUMAN; PRIORITY JOURNAL; RNA SPLICING;

ANIMALIA; MAMMALIA;

EID: 0029803547     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.93.23.12840     Document Type: Article

References (26)

1. Schwartz, E. & Benz, E. J. (1995) in Hematology, Basic Principles and Practice, eds. Huffman, R., Benz, E. J., Shattil, S. J., Furie, B. & Cohen, H. J. (Churchill Livingstone, New York), pp. 586-610.
2. Huisman, T. H. J. (1990) Br. J. Haematol. 75, 454-457.
3. Cheng, T.-C., Orkin, S. H., Antonorakis, S. E., Potter, M. J., Sexton, J. P., Giardina, P. J. V., Li, A. & Kazazian, H. H. (1984) Proc. Natl. Acad. Sci. USA 81, 2821-2826.
4. Huang, S.-Z., Zeng, F.-Y., Ren, Z.-R., Lu, Z.-H., Rodgers, G. P., Schechter, A. N. & Zeng, Y.-T. (1994) Br. J. Haematol. 88 541-546.
5. Busslinger, M. N., Moschonas, N. & Flavell, R. A. (1981) Cell 27, 289-296.
6. Fukumaki, Y., Ghosh, P. K., Benz, E. J., Reddy, V. B., Lebovitz, P., Forget, B. G. & Weissman, S. M. (1982) Cell 28, 585-594.
7. Treisman, R., Orkin, S. H. & Maniatis, T. (1983) Nature (London) 302, 591-595.
8. Dobkin, C. & Bank, A. (1985) J. Biol. Chem. 260, 16332-16337.
9. Dominski, Z. & Kole, R. (1993) Proc. Natl. Acad. Sci. USA 99, 8673-8677.
10. Crooke, S. T. & Lebleu, B., eds. (1993) Antisense Research and Applications (CRC, Boca Raton, FL).
11. Dominski, Z. & Kole, R. (1991) Mol. Cell. Biol. 11, 6075-6083.
12. Bennett, C. F., Chiang, M. Y., Chan, H., Shoemaker, J. & Mirabelli, C. K. (1992) Mol. Pharmacol. 41, 1023-1033.
13. Hawley-Nelson, P., Ciccarone, V., Gebeyehu, G., Jessee, J. & Felgner, P. L. (1993) Focus (Rochester, N.Y.) 15, 73-79.
14. Chen, I. T. & Chasin, L. A. (1993) Mol. Cell. Biol. 13, 289-300.
15. Schagger, H. & von Jagov, G. (1987) Anal. Biochem. 166, 368-379.
16. London, I. M., Clemens, M. J., Ranu, R. S., Levin, D. H., Cherbas. L. F. & Ernst, V. (1976) Fed. Proc. Fed. Am. Soc. Exp. Biol. 35, 2218-2222.
17. Sproat, B. S. & Lamond, A. I. (1993) in Antisense Research and Applications, eds. Crooke, S. T. & Lebleu, B. (CRC, Boca Raton, FL), pp. 351-363.
18. Furdon, P. F., Dominski, Z. & Kole, R. (1989) Nucleic Acids Res. 17, 9193-9204.
19. Fibach, E., Kollia, P., Schechter, A. N., Noguchi, C. T. & Rodgers, G. P. (1995) Blood 85, 2967-2974.
20. Weatherall, D. J. (1994) in The Molecular Basis of Blood Diseases, eds. Stamatoyannopoulos, G., Neinhuis, A. W., Leder, P. & Majerus, P. W. (Saunders, Philadelphia), pp. 157-205.
21. Green, M. R. (1991) Annu. Rev. Cell Biol. 7, 559-599.
22. Stein, C. A. (1995) Nat. Med. 1, 1119-1121.
23. Dominski, Z. & Kole, R. (1994) Mol. Cell. Biol. 14, 7445-7454.
24. Zhang, R., Lu, Z., Zhao, H., Zhang, X., Diasio, R. B., Habus, I., Jiang, Z., Iyer, R. P., Yu, D. & Agrawal, S. (1995) Biochem. Pharmacol. 50, 545-556.
25. Sher, G. D., Ginder, G. D., Little, J., Yang, S., Dover. G. J. & Olivieri, N. F. (1995) N. Engl. J. Med. 332, 1606-1610.
26. Voskaridou, E., Kalotychou, V. & Loukopoulos, D. (1995) Br. J. Haematol. 89, 479-484.