Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing

Journal of Clinical Investigation

Volumn 112, Issue 4, 2003, Pages 474-480

  Garcia Blanco, Mariano A ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GENE PRODUCT; MESSENGER RNA; TRANSMEMBRANE CONDUCTANCE REGULATOR;

CONSENSUS SEQUENCE; GENE STRUCTURE; GENETIC ANALYSIS; GENOME; HUMAN; PRIORITY JOURNAL; REVIEW; RNA PROCESSING; SPLICEOSOME; STRUCTURE ANALYSIS; TRANS SPLICING; ALTERNATIVE RNA SPLICING; ANIMAL; BIOLOGICAL MODEL; EXON; METABOLISM; MOLECULAR GENETICS; MUTATION; NUCLEOTIDE SEQUENCE; PHYSIOLOGY; RNA SPLICING;

ALTERNATIVE SPLICING; ANIMALS; BASE SEQUENCE; EXONS; HUMANS; MODELS, GENETIC; MOLECULAR SEQUENCE DATA; MUTATION; RNA SPLICING; RNA, MESSENGER; SPLICEOSOMES;

EID: 0141608626     PISSN: 00219738     EISSN: None     Source Type: Journal    
DOI: 10.1172/JCI200319462     Document Type: Review

References (31)

1. Dominski, Z., and Kole, R. 1993. Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. Proc. Natl. Acad. Sci. U. S. A. 90:8673-8677.
2. Kole, R., and Sazani, P. 2001. Antisense effects in the nucleus: modification of splicing. Curr. Opin. Mol. Ther. 3:229-234.
3. Sullenger, B.A., and Gilboa, E. 2002. Emerging clinical applications of RNA. Nature. 418:252-258.
4. Garcia-Blanco, M.A., Puttaraju, M., Mansfield, S.G., and Mitchell, L.G. 2000. Spliceosome-mediated RNA trans-splicing in gene therapy and genomics. Gene Ther Reg. 1:141-163.
5. Sullenger, B., and Cech, T.R. 1994. Ribozyme-mediated repair of defective mRNA by targeted trans-splicing. Nature. 317:619-622.
6. Lan, N., Howrey, R.P., Lee, S.W., Smith, C.A., and Sullenger, B. 1998. Ribozyme-mediated repair of sickly β-globin mRNAs in erythrocyte precursors. Science. 280:1593-1596.
7. Watanabe, T., and Sullenger, B. 2000. Induction of wild-type p53 activity in human cancer cells by ribozymes that repair mutant p53 transcripts. Proc. Natl. Acad. Sci. U. S. A. 97:8490-8494.
8. Ayre, B.G., Kohler, U., Goodman, H.M., and Haselhoff J. 1999. Design of highly specific cytotoxins by using trans-splicing ribozymes. Proc. Natl. Acad. Sci. U. S. A. 96:3507-3512.
9. Puttaraju, M., Jamison, S.F., Mansfield, S.G., Garcia-Blanco, M.A., and Mitchell, L.G. 1999. Spliceosome-mediated RNA trans-splicing as a tool for gene therapy. Nat. Biotech. 17:246-252.
10. Mansfield, S.G., et al. 2000. Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing. Gene Ther. 7:1885-1895.
11. Kikumori, T., Cote, G., and Gagel, R.F. 2001. Promiscuity of pre-mRNA spliceosome-mediated trans-splicing: a problem for gene therapy? Hum. Gene. Ther. 12:1429-1441.
12. Puttaraju, M., DiPasquale, J., Baker, C.C., Mitchell, L.G., and Garcia-Blanco, M.A. 2001. Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing. Mol. Ther. 4:105-114.
13. Liu, X., et al., 2002. Partial correction of endogenous ΔF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing. Nat. Biotech. 20:47-52.
14. Lander, E.S., et al. 2001. Initial sequencing and analysis of the human genome. Nature. 409:860-921.
15. Venter, J.C., et al. 2001. The sequence of the human genome. Science. 291:1304-1351.
16. Garcia-Blanco, M.A., Lindsey, L., and Ghosh, S. 2001. The phosphoryl transfer reactions in pre-messenger RNA splicing. In RNA. D. Soll, S. Nishimura, and P. Moore, edicors. Elsevier Science/Pergamon. Amsterdam, The Netherlands. 109-124.
17. Burge, C.B., Tuschl, T., and Sharp, P.A. 1999. Splicing of precursors to mRNAs by the spliceosomes. In The RNA world. R.F. Gesteland, T.R. Cech, and J.F. Atkins, editors. Cold Spring Harbor Laboratory Press. Cold Spring Harbor, New York, USA. 525-560.
18. Claverie, J.M. 2001. Gene number: what if there are only 30,000 human genes? Science. 291:1255-1257.
19. Crystal, R.C. 2002. Fooling mother nature. Nat. Biotech. 20:32-33.
20. Nilsen, T.W. 2001. Evolutionary origin of SL-addition trans-splicing: still an enigma. Trends Genet. 17:678-680.
21. Sullivan, P.M., Perrusz, P., Szpirer, C., and Joseph, D.R. 1991. Alternative processing of androgen-binding protein RNA transcripts in fetal rat liver. Identification of a transcript formed by trans splicing. J. Biol. Chem. 266:143-154.
22. Eul, J., Graessmann, M., and Graessmann, A. 1995. Experimental evidence for RNA trans-splicing in mammalian cells. EMBO J. 14:3226-3235.
23. Caudevilla, C., et al. 1998. Natural trans-splicing in carnitine octanoyl-transferase pre-mRNAs in rat liver. Proc. Natl. Acad. Sci. U. S. A. 95:12185-12190.
24. Tasic, B., et al. 2002. Promoter choice determines splice site selection in protocadherin alpha and gamma pre-mRNA splicing. Mol. Cell. 10:21-33.
25. Bruzik, J.P., and Maniatis, T. 1992. Spliced leader RNAs from lower eukaryotes are trans-spliced in mammalian cells. Nature. 360:692-695.
26. Pasman, Z., and Garcia-Blanco, M.A. 1996. The 5′ and 3′ splice sites come together via a three dimensional diffusion mechanism. Nucleic Acids Res. 24:1638-1645.
27. Goldstrohm, A.C., Greenleaf, A.L., and Garcia-Blanco, M.A. 2001. Cotranscriptional splicing of pre-messenger RNAs: considerations for the mechanism of alternative splicing. Gene. 277:31-47.
28. Maniatis, T., and Reed, R. 2002. An extensive network of coupling among gene expression machines. Nature. 416:499-506.
29. Konarska, M.M., Padgett, R.A., and Sharp, P.A. 1985. Trans-splicing of mRNA precursors in vitro. Cell. 42:165-171.
30. Solnick, D. 1985. Trans-splicing of mRNA precursors. Cell. 42:157-164.
31. Chao, H., et al. 2003. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing. Nat. Med. In press.