Therapeutic repair of mutated nucleic acid sequences

Nature Biotechnology

Volumn 16, Issue 4, 1998, Pages 341-344

  Woolf, Tod M ^a  

^a Sequitur Inc   (United States)

Author keywords

Antisense; Recombination; Ribozymes; RNA editing; Site directed mutagenesis; Splicing; Triple strand

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DOUBLE STRANDED DNA; RIBONUCLEASE H; RNA; SINGLE STRANDED DNA;

ALZHEIMER DISEASE; DNA RNA HYBRIDIZATION; DNA SEQUENCE; GENE MUTATION; GENE THERAPY; GENETIC DISORDER; NUCLEOTIDE SEQUENCE; POINT MUTATION; PRIORITY JOURNAL; REVIEW; RNA SEQUENCE;

DNA; DNA REPAIR; GENE THERAPY; HUMANS; MUTATION; RNA; RNA SPLICING;

EID: 0031970637     PISSN: 10870156     EISSN: None     Source Type: Journal    
DOI: 10.1038/nbt0498-341     Document Type: Review

References (19)

1. Woolf, T.W., Chase, J.M., and Stinchcomb, D. 1995. Towards the therapeutic editing of mutated RNA sequences. Proc. Natl. Acad. Sci, USA 92:8298-8302.
2. Sierakowska, H., Sambade, M.J., Agrawal, S., and Kole, R. 1996. Repair or thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides. Proc. Natl. Acad. Sci. USA 93:12840-12844.
3. Cole-Strauss, A., Yoon, K., Xiang, Y., Byrne, B.C., Rice, M.C., Kollman, W.K., and Kmiec, E.B. 1996. Correction of the mutation responsible for sickle cell anemia by an RNA-DNA Oligonucleotide. Science 273:1386-1389.
4. Jones, J.T., Seong-Wook, L., and Sullenger, B.A. 1996. Tagging ribozyme reaction sites to follow trans-splicing in mammalian cells. Nat. Med. 2:643-648.
5. Verma, I.M. and Somia, N. 1998. Gene therapy- promises, problems and prospects. Nature 389:239-242.
6. Vlassov, V., Gaidamakov, S., Zarytova, V., Knorre, D., Levina, A., Nikonova, A. et al. 1988. Sequence-specific chemical modification of double-stranded DNA with alkylating oligodeoxyribonucleotide derivatives. Gene 72:313-322.
7. Giovannangeli, C., Thuong, N., and Helene, C. 1992. Oligodeoxynucleotide-directed photo-induced cross-linking of HIV proviral DNA via triple-helix formation. Nucl. Acids Res. 20:4275-4281.
8. Wang, G., Levy, D., Seidman, M., and Glazer, P. 1995. Targeted mutagenesis in mammalian cells mediated by intracellular triple helix formation. Mol. Cell Biol. 15:1759-1568.
9. Vickers, T.A., Griffith, M.C., Ramasamy, K., Risen, L.M., and Freier, S.M. 1995. Inhibition of NF-kappa B specific transcriptional activation by PNA strand invasion. Nucl. Acids Res. 23:3003-3008.
10. Maher, L.d. and Dolnick, B.J. 1988. Comparative hybrid arrest by tandem antisense oligodeoxyribonucleotides or oligodeoxyribonucleoside methylphosphonates in a cell-free system. Nucl. Acids Res. 16:3341-3358.
11. Melton, D.A. 1985. Injected anti-sense RNAs specifically block messenger RNA translation in vivo. Proc. Natl. Acad. Sci. USA 82:144-148.
12. Lewin, B. 1990. Genes. Cell Press, Cambridge, UK.
13. Lukhtanoz, E.A., Mills, A.G., Kutyavin, I.V., Gorn, V.V., Reed, M.W., and Meyer, R.B. 1997. Minor groove DNA alkylation directed by major groove triplex-forming oligodeoxyribonucleotides. Nucl. Acids. Res. 25:5088-5084.
14. Bartel, D.P. and Szostak, J.W. 1993. Isolation of new ribozymes from a large pool of random sequences. Science 261:1411-1418.
15. Kren, B., Cole-Strauss, A., Kmiec, E., and Steer, C. 1997. Targeted nucleotide exchange in the alkaline phosphatase gene of HuH-7 cells mediated by a chimeric RNA/DNA oligonucleotide. Hepatology 25:1462-1468.
16. Yoon, K., Cole-Strauss, A., and Kmiec, E.B. 1996. Targeted gene correction of episomal DNA in mammalian cells mediated by a chimeric RNA:DNA oligonucleotide. Proc. Natl. Acad. Sci. USA 93:2071-2075.
17. Kren, B.T., Bandyopadhyay, P., and Steer, C.J. 1998. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides. Nat. Med. 4:285-290.
18. Dominski, Z. and Kole, R. 1993. Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. Proc. Natl. Acad. Sci. USA 90:8673-8677.
19. Sullenger, B. and Cech, T.R. 1994. Ribozyme-mediated repair of defective mRNA by targeted trans-splicing. Nature 371:619-622.