Pointing the finger at genetic disease

Gene Therapy

Volumn 12, Issue 19, 2005, Pages 1415-1416

  Cathomen, Toni ^a   Weitzman, M D ^b  

^a CHARITÉ UNIVERSITÄTSMEDIZIN BERLIN   (Germany)

^b SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DOUBLE STRANDED RNA; ENDONUCLEASE; EXTRACHROMOSOMAL DNA; INTERLEUKIN 2 RECEPTOR GAMMA; PARVOVIRUS VECTOR; ZINC FINGER PROTEIN; DEOXYRIBONUCLEASE;

BINDING SITE; CANCER RISK; DNA BINDING; DNA CLEAVAGE; DNA MODIFICATION; DNA REPAIR; DNA SEQUENCE; GENE EXPRESSION REGULATION; GENE INSERTION; GENE MUTATION; GENE SEQUENCE; GENE SILENCING; GENE TARGETING; GENE THERAPY; GENETIC DISORDER; GENOTOXICITY; HOMOZYGOSITY; HUMAN; HUMAN GENOME; IMMUNE DEFICIENCY; INFECTION PREVENTION; MAMMAL CELL; MUTAGENESIS; NONHUMAN; NOTE; PRIORITY JOURNAL; PROTEIN STRUCTURE; SEQUENCE ANALYSIS; VIRUS INHIBITION; X CHROMOSOME LINKAGE; ZINC FINGER MOTIF; ARTICLE; GENETICS; METABOLISM; METHODOLOGY;

MAMMALIA; PARVOVIRUS;

DNA REPAIR; ENDODEOXYRIBONUCLEASES; GENE TARGETING; GENE THERAPY; GENETIC DISEASES, INBORN; HUMANS; ZINC FINGERS;

EID: 27144447121     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302572     Document Type: Note

References (12)

1. Urnov FD et al. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 2005; advance online publication 3 April 2005; doi:10.1038/nature03556.
2. Hacein-Bey-Abina S et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348: 255-256.
3. Rouet P, Smih F, Jasin M. Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease. Mol Cell Biol 1994; 14: 8096-8106.
4. Beerli RR, Barbas III CF. Engineering polydactyl zinc-finger transcription factors. Nat Biotechnol 2002; 20: 135-141.
5. Reynolds L et al. Repression of the HIV-1 5′ LTR promoter and inhibition of HIV-1 replication by using engineered zinc-finger transcription factors. Proc Natl Acad Sci USA 2003; 100: 1615-1620.
6. Papworth M et al. Inhibition of herpes simplex virus 1 gene expression by designer zinc-finger transcription factors. Proc Natl Acad Sci USA 2003; 100: 1621-1626.
7. Tan W et al. Fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc finger protein E2C direct integration of viral DNA into specific sites. J Virol 2004; 78: 1301-1313.
8. Bibikova M et al. Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol Cell Biol 2001; 21 289-297.
9. Kim YG, Cha J, Chandrasegaran S. Hybrid restriction enzymes: Zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci USA 1996; 93: 1156-1160.
10. Smith J et al. Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains. Nucleic Acids Res 2000; 28: 3361-3369.
11. Porteus MH, Cathomen T, Weitzman MD, Baltimore D. Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks. Mol Cell Biol 2003; 23: 3558-3565.
12. Miller DG, Petek LM, Russell DW. Human gene targeting by adeno-associated virus vectors is enhanced by DNA double-strand breaks. Mol Cell Biol 2003; 23: 3550-3557.