Spliceosome-mediated RNA trans-splicing

Molecular Therapy

Volumn 12, Issue 6, 2005, Pages 1006-1012

  Yang, Yanping ^a   Walsh, Christopher E ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Gene transfer; RNA trans splicing; Spliceosome; Vector

Indexed keywords

ADENOVIRUS VECTOR; APOLIPOPROTEIN A1; BLOOD CLOTTING FACTOR 8; CD40 LIGAND; COBROTOXIN; COMPLEMENTARY DNA; ENDONUCLEASE; LENTIVIRUS VECTOR; MESSENGER RNA PRECURSOR; NAKED DNA; NEOMYCIN; PLASMID DNA; PROTEIN E6; PROTEIN E7; RIBOZYME; TRANSFER RNA; TRANSMEMBRANE CONDUCTANCE REGULATOR; VEROTOXIN 1;

AUTOSOMAL DOMINANT DISORDER; CARDIOVASCULAR DISEASE; COMBINED IMMUNODEFICIENCY; DIAGNOSTIC TEST; GENE TECHNOLOGY; GENE THERAPY; GENETIC DISORDER; HEMOPHILIA A; IN VIVO STUDY; LUNG FIBROSIS; MOLECULAR BIOLOGY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PHENOTYPE; REVIEW; RNA SPLICING; SPLICEOSOME; SUICIDE GENE THERAPY; TRANS SPLICING; UTERINE CERVIX CANCER; VIRAL GENE DELIVERY SYSTEM; X CHROMOSOME LINKED DISORDER;

ANIMALS; DNA REPAIR; GENE THERAPY; HUMANS; MICE; MODELS, BIOLOGICAL; RNA; RNA SPLICING; RNA, CATALYTIC; RNA, MESSENGER; RNA, TRANSFER; SPLICEOSOMES;

ADENOVIRIDAE; LENTIVIRUS;

EID: 28444453994     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.09.006     Document Type: Review

References (41)

1. C.E. Walsh New paradigm for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategies Semin. Hematol. 41 2004 297-302
2. S.G. Mansfield H. Chao and C.E. Walsh RNA repair using spliceosome-mediated RNA trans-splicing Trends Mol. Med. 10 2004 263-268
3. M.A. Garcia-Blanco Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing J. Clin. Invest. 112 2003 474-480
4. P. Sazani and R. Kole Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing J. Clin. Invest. 112 2003 481-486
5. B.A. Sullenger Targeted genetic repair: An emerging approach to genetic therapy J. Clin. Invest. 112 2003 310-311
6. N.A. Faustino and T.A. Cooper Pre-mRNA splicing and human disease Genes Dev. 17 2003 419-437
7. M.A. Garcia-Blanco A.P. Baraniak and E.L. Lasda Alternative splicing in disease and therapy Nat. Biotechnol. 22 2004 535-546
8. W.J. Murphy K.P. Watkins and N. Agabian Identification of a novel Y branch structure as an intermediate in trypanosome mRNA processing: evidence for trans splicing Cell 47 1986 517-525
9. A. Rajkovic R.E. Davis J.N. Simonsen and F.M. Rottman A spliced leader is present on a subset of mRNAs from the human parasite Schistosoma mansoni Proc. Natl. Acad. Sci. USA 87 1990 8879-8883
10. R. Sutton and J. Boothroyd Evidence for trans-splicing in trypanosomes Cell 47 1986 527-535
11. J. Bruzik and T. Maniatis Spliced leader RNAs from lower eukaryotes are trans-spliced in mammalian cells Nature 360 1992 692-695
12. C. Caudevilla et al. Natural trans-splicing in carnitine octanoyltransferase pre-mRNAs in rat liver Proc. Natl. Acad. Sci. USA 95 1998 12185-12190
13. C. Finta and P. Zaphiropoulos Intergenic mRNA molecules resulting from trans-splicing J. Biol. Chem. 277 2002 5882-5890
14. G. Flouriot H. Brand B. Seraphin and F. Gannon Natural trans-spliced mRNAs are generated from the human estrogen receptor- (hER) gene J. Biol. Chem. 277 2002 26244-26251
15. R. Dorn and V. Krauss The modifier of mdg4 locus in Drosophila: functional complexity is resolved by trans-splicing Genetica 117 2003 165-177
16. M. Labrador and V. Corces Extensive exon reshuffling over evolutionary time coupled to trans-splicing in Drosophila Genome Res. 13 2003 2220-2228
17. M. Puttaraju S.F. Jamison S.G. Mansfield M.A. Garcia-Blanco and L.G. Mitchell Spliceosome-mediated RNA trans-splicing as a tool for gene therapy Nat. Biotechnol. 17 1999 246-252
18. B.A. Sullenger and E. Gilboa Emerging clinical applications of RNA Nature 418 2002 252-258
19. G. Dallinger et al. Development of spliceosome-mediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases Exp. Dermatol. 12 2003 37-46
20. S.G. Mansfield et al. 5′ Exon replacement and repair by spliceosome-mediated RNA trans-splicing RNA 9 2003 1290-1297
21. M. Puttaraju J. DiPasquale C.C. Baker L.G. Mitchell and M.A. Garcia-Blanco Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing Mol. Ther. 4 2001 105-114
22. S.G. Mansfield et al. Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing Gene Ther. 7 2000 1885-1895
23. X. Liu et al. Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing Nat. Biotechnol. 20 2002 47-52
24. H. Chao et al. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing Nat. Med. 9 2003 1015-1019
25. B.A. Sullenger and T.R. Cech Ribozyme-mediated repair of defective mRNA by targeted trans-splicing Nature 371 1994 619-622
26. M.B. Long J.P. Jones III B.A. Sullenger and J. Byun Ribozyme-mediated revision of RNA and DNA J. Clin. Invest. 112 2003 312-318
27. G. Deidda N. Rossi and G.P. Tocchini-Valentini An archaeal endoribonuclease catalyzes cis- and trans-nonspliceosomal splicing in mouse cells Nat. Biotechnol. 21 2003 1499-1504
28. L. Bi A.M. Lawler S.E. Antonarakis K.A. High J.D. Gearhart and H.H. Kazazian Jr. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A Nat. Genet. 10 1995 119-121
29. M. Tahara N. Hackett R.G. Pergolizzi and R. Crystal Trans-splicing-mediated CD40-ligand gene therapy correction of murine model of correction human X-linked immunodeficiency disease Mol. Ther. 7 2003 S154
30. R.G. Pergolizzi et al. 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 999-1008
31. S. Bhaumik Z. Walls M. Puttaraju L.G. Mitchell and S.S. Gambhir Molecular imaging of gene expression in living subjects by spliceosome-mediated RNA trans-splicing Proc. Natl. Acad. Sci. USA 101 2004 8693-8698
32. T. Kikumori G.J. Cote and R.F. Gagel Promiscuity of pre-mRNA spliceosome-mediated trans-splicing: A problem for gene therapy? Hum Gene Ther. 12 2001 1429-1441
33. E. Wagner and J. Lykke-Andersen mRNA surveillance: The perfect persist J. Cell Sci. 115 2002 3033-3038
34. B. Seed and A. Aruffo Molecular cloning of CD2 antigen, the T-cell erythrocyte receptor, by a rapid immunoselection procedure Proc. Natl. Acad. Sci. USA 84 1987 3365-3369
35. D. Solnick Trans-splicing of mRNA precursors Cell 42 1985 157-164
36. M.M. Konarska R.A. Padgett and P.A. Sharp Trans-splicing of mRNA precursors in vitro Cell 42 1985 165-171
37. M. Patrick et al. Optimizing the efficacy of spliceosome-mediated RNA trans-splicing for suicide gene therapy of cervical cancer Mol. Ther. 11 2005 S115
38. M. Puttaraju et al. Spliceosome mediated RNA trans-splicing strategy to increase the production of human apolipoprotein (ApoA1) Mol. Ther. 11 2005 S429
39. K. Nakayama R.G. Pergolizzi and R.G. Crystal Gene transfer-mediated pre-mRNA segmental trans-splicing as a strategy to deliver intracellular toxins for cancer therapy Cancer Res. 65 2005 254-263
40. R. Kornblihtt et al. Multiple links between transcription and splicing RNA 10 2004 1489-1498
41. A.S. Phillip The discovery of split genes and RNA splicing Trends Biochem. Sci. 30 2005 279-281