Gene repair using chimeric RNA/DNA oligonucleotides

Seminars in Liver Disease

Volumn 19, Issue 1, 1999, Pages 93-104

  Kren, Betsy T ^a   Metz, Richard ^b   Kumar, Ramesh ^b   Steer, Clifford J ^a,c,d  

^a Department of Medicine   (United States)

^b Kimeragen   (United States)

^c UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^d UNIV OF MINNESOTA HOSP AND CLINIC   (United States)

Author keywords

Asialoglycoprotein receptor; Chimeraplasty; Gene therapy; Homologous recombination; Mismatch repair; Nucleotide exchange; Polyethylenimine

Indexed keywords

ASIALOGLYCOPROTEIN RECEPTOR; CARBONATE DEHYDRATASE II; COMPLEMENTARY DNA; COMPLEMENTARY RNA; OLIGONUCLEOTIDE; POLYETHYLENEIMINE;

BASE MISPAIRING; CHIMERA; DNA REPAIR; DNA RNA HYBRIDIZATION; DNA SEQUENCE; GENE THERAPY; GENETIC DISORDER; HEPATOMA CELL; HUMAN; LIVER CELL; LIVER DISEASE; MISSENSE MUTATION; NONHUMAN; NONSENSE MUTATION; POINT MUTATION; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; REVIEW;

EID: 0032945304     PISSN: 02728087     EISSN: None     Source Type: Journal    
DOI: 10.1055/s-2007-1007101     Document Type: Review

References (44)

1. Yáñez RJ, Porter ACG. Therapeutic gene targeting. Gene Ther 1998;5:149-159
2. Kotani H, Kmiec EB. A role for RNA synthesis in homologous pairing events. Mol Cell Biol 1994;14:6097-6106
3. Kotani H, Kmiec EB. Transcription activates RecA-promoted homologous pairing of nucleosomal DNA. Mol Cell Biol 1994;14:1949-1955
4. Thomas KR, Folger KR, Capecchi MR. High frequency targeting of genes to specific sites in the mammalian genome. Cell 1986;44:419-428
5. Smithies O, Gregg RG, Boggs SS, Koralewski MA, Kucherlapati RS. Insertion of DNA sequences into the human chromosomal β-globin locus by homologous recombination. Nature 1985;317:230-234
6. Roth DB, Wilson JH. Relative rates of homologous and nonhomologous recombination in transfected DNA. Proc Natl Acad Sci U S A 1985;82:3355-3359
7. Rubnitz J, Subramani S. The minimum amount of homology required for homologous recombination in mammalian cells. Mol Cell Biol 1984;4:2253-2258
8. Yoon K, Cole-Strauss A, Kmiec EB. Targeted gene correction of episomal DNA in mammalian cells mediated by a chimeric RNA•DNA oligonucleotide. Proc Natl Acad Sci U S A 1996;93:2071-2076
9. Russell DW, Hirata RK. Human gene targeting by viral vectors. Nat Genet 1998;18:325-330
10. Roth D, Wilson J. Illegitimate recombination in mammalian cells. In: Kucherlapati R, Smith GR (eds). Genetic Recombination. Washington, DC: ASM Press, 1988, pp 621-653
11. Metz R, DiCola M, Primerano J, Thomson N, Dhanjal N, Avissar P, Kumar R. Mismatch directed repair mechanism for in vivo targeted mutagenesis in Escherichia coli employing synthetic RNA/DNA oligonucleotides (submitted for publication).
12. Modrich P, Lahue R. Mismatch repair in replication fidelity, genetic recombination, and cancer biology. Ann Rev Biochem 1996;65:101-133
13. Thomas KR, Capecchi MR. Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells. Cell 1987;51: 503-512
14. Wang G, Levy DD, Seidman MM, Glazer PM. Targeted mutagenesis in mammalian cells mediated by intracellular triple helix formation. Mol Cell Biol 1995;15:1759-1768
15. Beal PA, Dervan PB. Second structural motif for recognition of DNA by oligonucleotide-directed triple-helix formation. Science 1991;251:1360-1363
16. Sakagami K, Tokinaga Y, Yoshikura H, Kobayashi I. Homology-associated nonhomologous recombination in mammalian gene targeting. Proc Natl Acad Sci U S A 1994;91:8527-8531
17. Cole-Strauss A, Yoon K, Xiang Y, et al. Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide. Science 1996;273:1386-1389
18. Kren BT, Cole-Strauss A, Kmiec EB, Steer CJ. Targeted nucleotide exchange in the alkaline phosphatase gene of HuH-7 cells mediated by a chimeric RNA/DNA oligonucleotide. Hepatology 1997;25:1462-1468
19. Kishi F, Matsuura S, Kajii T. Nucleotide sequence of the human liver-type alkaline phosphatase cDNA. Nucleic Acids Res 1989;17:2129
20. Abdallah B, Hassan A, Benoist C, Goula D, Behr JP. Demeneix BA. A powerful nonviral vector for in vivo gene transfer into the adult mammalian brain: polyethylenimine. Hum Gene Ther 1996;7:1947-1954
21. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B, Behr J-P. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: Polyethylenimine. Proc Natl Acad Sci U S A 1995;92:7297-7301
22. Weiss MJ, Ray K, Henthorn PS, Lamb B, Kadesch T, Harris H. Structure of the human liver/bone/kidney alkaline phosphatase gene. J Biol Chem 1988;263:12002-12010
23. Kotani H, Germann MW, Andrus A, Vinayak R, Mullah B, Kmiec EB. RNA facilitates RecA-mediated DNA pairing and strand transfer between molecules bearing limited regions of homology. Mol Gen Genet 1996;250:626-634
24. Fox IJ, Chowdhury JR, Kaufman SS, et al. Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation. N Engl J Med 1998;338:1422-1426
25. Perales JC, Ferkol T, Beegen H, Ratnoff OD, Hanson RW. Gene transfer in vivo: Sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc Natl Acad Sci U S A 1994;91:4086-4090
26. Findeis MA, Wu CH, Wu GY. Ligand-based carrier systems for delivery of DNA to hepatocytes. Methods Enzymol 1994;247: 341-351
27. Gray GR. The direct coupling of oligosaccharides to proteins and derivatized gels. Arch Biochem Biophys 1974;163:426-428
28. Bandyopadhyay P, Kren BT, Ma X, Steer CJ. Enhanced gene transfer into HuH-7 cells and primary rat hepatocytes using targeted liposomes and polyethylenimine. Biotechniques 1998;25: 282-292
29. Spanjer HH, Scherphof GL. Targeting of lactosylceramide-containing liposomes to hepatocytes in vivo. Biochim Biophys Acta 1983;734:40-47
30. Nandi PK, Legrand A, Nicolau C. Biologically active, recombinant DNA in clathrin-coated vesicles isolated from rat livers after in vivo injection of liposome-encapsulated DNA. J Biol Chem 1986;261:16722-16726
31. Templeton NS, Lasic DD, Frederik PM, Strey HH, Roberts DD, Pavlakis GN. Improved DNA: liposome complexes for increased systemic delivery and gene expression. Nature Biotechnol 1997;15:647-652
32. Kren BT, Bandyopadhyay P, Steer CJ. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides. Nat Med 1998;4:285-290
33. Sarkar G, Koeberl DD, Sommer SS. Direct sequencing of the activation peptide and the catalytic domain of the factor IX gene in six species. Genomics 1990;6:133-143
34. Boletta A, Benigni A, Lutz J, Remuzzi G, Soria MR, Monaco L. Nonviral gene delivery to the rat kidney with polyethylenimine. Hum Gene Ther 1997;8:1243-1251
35. Higgins GM, Anderson RM. Experimental pathology of the liver. I. Restoration of the liver of the white rat following partial surgical removal. Arch Pathol 1931;12;186-202
36. Askari FK, Hitomi Y, Mao M, Wilson JM. Complete correction of hyperbilirubinemia in the Gunn rat model of Crigler-Najjar syndrome type I following transient in vivo adenovirus-mediated expression of human bilirubin UDP-glucuronosyltransferase. Gene Ther 1996;3:381-388
37. Ilan Y, Prakash R, Davidson A, et al. Oral tolerization to adenoviral antigens permits long-term gene expression using recombinant adenoviral vectors. J Clin Invest 1997;99:1098-1106
38. Wellinger RE, Thoma F. Nucleosome structure and positioning modulate nucleotide excision repair in the non-transcribed strand of an active gene. EMBO J 1997;16:5046-5056
39. Klungland A, Lindahl T. Second pathway for completion of human DNA base excision-repair: reconstitution with purified proteins and requirement for DNase IV (FEN1). EMBO J 1997; 16:3341-3348
40. Fang W-h, Modrich P. Human strand-specific mismatch repair occurs by a bidirectional mechanism similar to that of the bacterial reaction. J Biol Chem 1993;268:11838-11844
41. 1-antitrypsin gene expression in somatic cell hybrids: evidence for multiple controls. Genes Dev 1992;6:316-327
42. Lai L-W, O'Connor HM, Lien Y-H. Correction of carbonic anhydrase II mutation in renal tubular cells by chimeric RNA/DNA oligonucleotide. Conference Proceedings: 1st Annual Meeting of the American Society of Gene Therapy, Seattle, WA, 1998, p 183a
43. Alexeev V, Yoon K. Stable and inheritable changes in genotype and phenotype of albino melanocytes induced by an RNA-DNA oligonucleotide. Nature Biotech 1998;16:1343-1346
44. Bartlett RJ, Denis MM, Kornegay JN, et al. Can genetic surgery be used to revert muscular dystrophy mutations in live animals? Conference Proceedings: 1st Annual Meeting of the American Society of Gene Therapy, Seattle, WA, 1998, p 153a