Engineered zinc finger proteins for manipulation of the human mitochondrial genome

Methods in Molecular Biology

Volumn 649, Issue , 2010, Pages 257-270

  Minczuk, Michal ^a  

^a MEDICAL RESEARCH COUNCIL   (United Kingdom)

Author keywords

engineered zinc fingers; gene therapy; mitochondrial diseases; Mitochondrial import; nuclear export signal; zinc finger nuclease

Indexed keywords

MAMMALIA;

ENDONUCLEASE; ZINC FINGER PROTEIN;

ARTICLE; DISORDERS OF MITOCHONDRIAL FUNCTIONS; FLUORESCENT ANTIBODY TECHNIQUE; GENE THERAPY; GENETICS; HUMAN; METABOLISM; METHODOLOGY; MITOCHONDRIAL GENOME; PROTEIN ENGINEERING; TUMOR CELL LINE;

CELL LINE, TUMOR; ENDONUCLEASES; FLUORESCENT ANTIBODY TECHNIQUE; GENE THERAPY; GENOME, MITOCHONDRIAL; HUMANS; MITOCHONDRIAL DISEASES; PROTEIN ENGINEERING; ZINC FINGERS;

EID: 80054904545     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-60761-753-2_16     Document Type: Article

References (21)

1. Taylor, R.W. and Turnbull, D.M. (2005) Mitochondrial DNA mutations in human disease. Nat Rev Genet. 6, 389-402.
2. Schapira, A.H. (2006) Mitochondrial disease. Lancet. 368, 70-82.
3. Tang, Y., Manfredi, G., Hirano, M., and Schon, E.A. (2000) Maintenance of human rearranged mitochondrial DNAs in longterm cultured transmitochondrial cell lines. Mol Biol Cell. 11, 2349-2358.
4. Taylor, R.W., Chinnery, P.F., Turnbull, D.M., and Lightowlers, R.N. (1997) Selective inhibition of mutant human mitochondrial DNA replication in vitro by peptide nucleic acids. Nat Genet. 15, 212-215.
5. Tanaka, M., Borgeld, H.J., Zhang, J., Muramatsu, S., Gong, J.S., Yoneda, M., Maruyama,W., Naoi, M., Ibi, T., Sahashi, K., et al. (2002) Gene therapy for mitochondrial disease by delivering restriction endonuclease SmaI into mitochondria. J Biomed Sci. 9, 534-541.
6. Srivastava, S. and Moraes, C.T. (2001) Manipulating mitochondrial DNA heteroplasmy by a mitochondrially targeted restriction endonuclease. Hum Mol Genet. 10, 3093-3099.
7. Alexeyev,M.F., Venediktova, N., Pastukh, V., Shokolenko, I., Bonilla, G., and Wilson, G.L. (2008) Selective elimination of mutant mitochondrial genomes as therapeutic strategy for the treatment of NARP and MILS syndromes. Gene Ther. 15, 516-523.
8. Bayona-Bafaluy, M.P., Blits, B., Battersby, B.J., Shoubridge, E.A., and Moraes, C.T. (2005) Rapid directional shift of mitochondrial DNA heteroplasmy in animal tissues by a mitochondrially targeted restriction endonuclease. Proc Natl Acad Sci USA. 102, 14392-14397.
9. Bacman, S.R.,Williams, S.L., Hernandez, D., and Moraes, C.T. (2007) Modulating mtDNA heteroplasmy by mitochondriatargeted restriction endonucleases in a 'differential multiple cleavage-site' model. Gene Ther. 14, 1309-1318.
10. Krishnan, K.J., Reeve, A.K., Samuels, D.C., Chinnery, P.F., Blackwood, J.K., Taylor, R.W., Wanrooij, S., Spelbrink, J.N., Lightowlers, R.N., and Turnbull, D.M. (2008) What causes mitochondrial DNA deletions in human cells? Nat Genet. 40, 275-279.
11. Papworth, M., Kolasinska, P., and Minczuk, M. (2006) Designer zinc-finger proteins and their applications. Gene. 366, 27-38.
12. Kim, Y.G., Cha, J., and Chandrasegaran, S. (1996) Hybrid restriction enzymes: Zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci USA. 93, 1156-1160.
13. Minczuk, M., Papworth, M.A., Kolasinska, P., Murphy, M.P., and Klug, A. (2006) Sequence-specific modification of mitochondrial DNA using a chimeric zinc finger methylase. Proc Natl Acad Sci USA. 103, 19689-19694.
14. Minczuk, M., Papworth, M.A., Miller, J.C., Murphy, M.P., and Klug, A. (2008) Development of a single-chain, quasi-dimeric zinc-finger nuclease for the selective degradation of mutated human mitochondrial DNA. Nucleic Acids Res. 36, 3926-3938.
15. Fernandez-Martinez, J., Brown, C.V., Diez, E., Tilburn, J., Arst, H.N., Jr., Penalva, M.A., and Espeso, E.A. (2003) Overlap of nuclear localisation signal and specific DNA-binding residues within the zinc finger domain of PacC. J Mol Biol. 334, 667-684.
16. Matheny, C., Day, M.L., and Milbrandt, J. (1994) The nuclear localization signal of NGFI-A is located within the zinc finger DNA binding domain. J Biol Chem. 269, 8176-8181.
17. Papworth, M., Moore, M., Isalan, M., Minczuk, M., Choo, Y., and Klug, A. (2003) Inhibition of herpes simplex virus 1 gene expression by designer zinc-finger transcription factors. Proc Natl Acad Sci USA. 100, 1621-1626.
18. Pfanner, N. and Geissler, A. (2001) Versatility of the mitochondrial protein import machinery. Nat Rev Mol Cell Biol. 2, 339-349.
19. Gaines, G.L., 3rd.. (1996) In organello RNA synthesis system from HeLa cells. Methods Enzymol. 264, 43-49.
20. Hartmann, C.M., Gehring, H., and Christen, P. (1993) The mature form of imported mitochondrial proteins undergoes conformational changes upon binding to isolated mitochondria. Eur J Biochem. 218, 905-910.
21. Taylor, S.W., Fahy, E., Zhang, B., Glenn, G.M., Warnock, D.E., Wiley, S., Murphy, A.N., Gaucher, S.P., Capaldi, R.A., Gibson, B.W., et al. (2003) Characterization of the human heart mitochondrial proteome. Nat Biotechnol. 21, 281-286.