New paradigms for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategies

Seminars in Hematology

Volumn 41, Issue 4 SPEC.ISS., 2004, Pages 297-302

  Walsh, Christopher E ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; COMPLEMENTARY DNA; ENDONUCLEASE; MESSENGER RNA; SMALL INTERFERING RNA;

GENE THERAPY; GENE TRANSFER; HUMAN; NONHUMAN; NUCLEIC ACID STRUCTURE, METABOLISM AND FUNCTION; PRIORITY JOURNAL; REVIEW; RNA REPAIR; RNA SPLICING; SPLICEOSOME; TRANS SPLICING;

EID: 7044227854     PISSN: 00371963     EISSN: None     Source Type: Journal    
DOI: 10.1053/j.seminhematol.2004.07.006     Document Type: Article

References (28)

1. T. Abbas-Terki, W. Blanco-Bose, N. Deglon, W. Pralong, P. Aebischer Lentiviral-mediated RNA interference Hum Gene Ther 13 2002 2197
2. J. Kowalski, V. Derbyshire Characterization of homing endonucleases Methods 28 2002 365
3. P. Sazani, R. Kole Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing J Clin Invest 112 2003 481
4. W.J. Murphy, K.P. Watkins, N. Agabian Identification of a novel Y branch structure as an intermediate in trypanosome mRNA processing Evidence for trans splicing Cell 47 1986 517
5. A. Rajkovic, R. Davis, J. Simonsen, F. Rottman A spliced leader is present on a subset of mRNAs from the human parasite Schistosoma mansoni Proc Natl Acad Sci USA 87 1990 8879
6. C. Caudevilla, D. Serra, A. Miliar, C. Codony, G. Asins, M. Bach Natural trans-splicing in carnitine octanoyltransferase pre-mRNAs in rat liver Proc Natl Acad Sci USA 95 1998 12185
7. C. Finta, P. Zaphiropoulos Intergenic mRNA molecules resulting from trans-splicing J Biol Chem 277 2002 5882
8. A. Romani, E. Guerra, M. Trerotola, S. Alberti Detection and analysis of spliced chimeric mRNAs in sequence databanks Nucleic Acids Res 31 2003 e17
9. M. Labrador, V. Corces Extensive exon reshuffling over evolutionary time coupled to trans-splicing in Drosophila Genome Res 13 2003 2220
10. L. Bi, A.M. Lawler, S.E. Antonarakis, K.A. High, J.D. Gearhart, H.H. Kazazian Jr Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A Nat Genet 10 1995 119
11. X. Liu, Q. Jiang, S.G. Mansfield, M. Puttaraju, Y. Zhang, W. Zhou Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing Nat Biotechnol 20 2002 47
12. M. Puttaraju, S. Jamison, S. Mansfield, M. Garcia-Blanco, L. Mitchell Spliceosome-mediated RNA trans-splicing as a tool for gene therapy Nat Biotechnol 17 1999 246
13. M. Puttaraju, J. DiPasquale, C.C. Baker, L.G. Mitchell, M.A. Garcia-Blanco Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing Mol Ther 4 2001 105
14. G. Dallinger, M. Puttaraju, L.G. Mitchell, K. Yancey, C. Yee, A. Klausegger Development of spliceosome-mediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases Exp Dermatol 12 2003 37
15. M. Garcia-Blanco Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing J Clin Invest 112 2003 474
16. E. Otto, G. Temple, G. McGarrity Re-programming gene expression using SMaRT Curr Drug Discovery ? 2003 37
17. H. Chao, S.G. Mansfield, R.C. Bartel, S. Hiriyanna, L.G. Mitchell, M.A. Garcia-Blanco Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing Nat Med 9 2003 1015
18. J. Kolkman, W. Stemmer Directed evolution of proteins by exon shuffling Nat Biotechnol 19 2001 423
19. S. Mansfield, J. Kole, M. Puttaraju, C. Yang, M. Garcia-Blanco, J. Cohn Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing Gene Ther 7 2000 1885
20. M. Tahara, N. Hackett, R. Pergolozzi, R. Crystal Trans-splicing-mediated CD40-ligand gene therapy correction of a murine model of correction human X-linked immunodeficiency disease Mol Ther 7 2003 S154
21. R. Pergolizzi, A. Ropper, R. Dragos, A. Reid, K. Nakayama, Y. Tan In vivo trans-splicing of 5′ and 3′ segments of pre-mRNA directed by corresponding DNA sequences delivered by gene transfer Mol Ther 8 2003 999
22. T. Kikumori, G.J. Cote, R.F. Gagel Promiscuity of pre-mRNA spliceosome-mediated trans splicing A problem for gene therapy? Hum Gene Ther 12 2001 1429
23. S. Mansfield, R. Clark, M. Puttaraju, J. Kole, J. Cohn, L. Mitchell 5′ Exon replacement and repair by spliceosome-mediated RNA trans-splicing RNA 9 2003 1290
24. Y. Shi Mammalian RNAi for the masses Trends Genet 19 2003 9
25. M. Tijsterman, R. Ketting, R. Plasterk The genetics of RNA silencing Ann Rev Genet 36 2002 489
26. M. Hemann, J. Fridman, J. Zilfou, E. Hernando, P. Paddison, C. Cordon-Cardo An epi-allelic series of p53 hypomorphs created by stable RNAi produces distinct tumor phenotypes in vivo Nat Genet 33 2003 396
27. M. Scherr, K. Battmer, T. Winkler, O. Heidenreich, A. Ganser, M. Eder Specific inhibition of bcr-abl gene expression by small interfering RNA Blood 101 2003 1566
28. H. Arteaga, J. Hinkula, I. van Dijk-Hard, M. Dilber, B. Wahren, B. Christensson Choosing CCR5 or Rev siRNA in HIV-1 Nat Biotechnol 21 2003 230