A crisper look at genome editing: RNA-guided genome modification

Molecular Therapy

Volumn 21, Issue 4, 2013, Pages 720-722

  Damian, Mara ^a   Porteus, Matthew H ^a  

^a STANFORD UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DEOXYRIBONUCLEASE; DNA; NUCLEASE; RNA; TAL EFFECTOR NUCLEASE; TYPE II SITE SPECIFIC DEOXYRIBONUCLEASE; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE;

ADAPTIVE IMMUNITY; ADENO ASSOCIATED VIRUS; CLUSTERED REGULATORY INTERSPACED SHORT PALINDROMIC REPEATS; DIMERIZATION; DNA BINDING; DNA MODIFICATION; DNA SEQUENCE; GENETIC ASSOCIATION; IMMUNE SYSTEM; NONHUMAN; NOTE; PROTEIN ENGINEERING;

EID: 84878594015     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.46     Document Type: Note

References (20)

1. Cong, L, Ran, FA, Cox, D, Lin, S, Barretto, L, Habib, N et al. (2013). Multiplex genome engineering using CRISPR/Cas Systems. Science, e-pub ahead of print 3 January 2013.
2. Mali, P, Yang, L, Esvelt, KM, Aach, J, Guell, M, Di-Carlo, JE et al. (2013). RNA-guided human genome engineering via Cas9. Science, e-pub ahead of print 3 January 2013.
3. Stoddard, BL (2011). Homing endonucleases: from microbial genetic invaders to reagents for targeted DNA modifcation. Structure 19: 7-15.
4. Urnov, FD, Miller, JC, Lee, YL, Beausejour, CM, Rock, JM, Augustus, S et al. (2005). Highly effcient endogenous human gene correction using designed zinc-fnger nucleases. Nature 435: 646-651.
5. Miller, JC, Tan, S, Qiao, G, Barlow, KA, Wang J, Xia, DF et al. (2011). A TALE nuclease architecture for effcient genome editing. Nat Biotechnol 29: 143-150.
6. Aiba, Y, Sumaoka, J and Komiyama, M (2011). Artifcial DNA cutters for DNA manipulation and genome engineering. Chem Soc Rev 40: 5657-5668.
7. Hendrie, PC, Hirata RK and Russell, DW (2003). Chromosomal integration and homologous gene targeting by replication-incompetent vectors based on the autonomous parvovirus minute virus of mice. J Virol 77: 13136-13145.
8. Jurica, MS and Soddard, BL (1999). Homing endo-nucleases: structure, function and evolution. Cell Mol Life Sci 10: 1304-1326.
9. Russell, DW and Hirata, RK (1998). Human gene targeting by viral vectors. Nat Genet 4: 325-330.
10. Kim, YG, Cha, Y and Chandrasegaram, S (1996). Hybrid restriction enzymes: zinc fnger fusion to FokI cleavage domain. Proc Natl Acad Sci USA 93: 1156-1160.
11. Bibikova, M, Beumer, K, Trautman, JK and Carroll, D (2003). Enhancing gene targeting with designed zinc fnger nucleases. Science 300: 764.
12. Porteus, MH and Baltimore, D (2003). Chimeric nucleases stimulate gene targeting in human cells. Science 300: 763
13. + T cells by genome editing using zinc-fnger nucleases. Nat Biotechnol 26: 808-816.
14. Komiyama, M, Aiba, Y, Yamamoto, Y and Sumaoka, J (2008). Artifcial restriction DNA cutter for site-selective scission of double-stranded DNA with tunable scission site and specifcity. Nat Protoc 4: 655-662.
15. Bogdanove, AJ and Voytas, DF (2011). TAL effectors: customizable proteins for DNA targeting. Science 6051: 1843-1846.
16. Pruett-Miller, SM, Connelly, JP, Maeder, ML, Joung, JK and Porteus, MH (2008). Comparison of zinc fnger nucleases for use in gene targeting in mammalian cells. Mol Ther 16: 707-717.
17. Jinek, M, Chylinski, K, Fonfara, I, Hauer, M, Doud-na, JA and Charpentier, E (2012). A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337: 816-821.
18. Weinstock, DM, Nakanishi, K, Helgadottir, HR and Jasin, M (2006). Assaying double-strand break repair pathway choice in mammalian cells using a targeted endonuclease or the RAG recombinase. Methods Enzymol 409: 524-540.
19. Ramirez, CL, Certo, MT, Mussolino, C, Goodwin, MJ, Cradick, TJ, McCaffrey, AP et al. (2012). Engineered zinc fnger nickases induce homologous-direct repair with reduced mutagenic effects. Nucleic Acids Res 40: 5560-5568.
20. Wang, J, Friedman, G, Doyon, Y, Wang, NS, Li, CJ, Miller, JC et al. (2012). Targeted gene addition to a predetermined site in the human genome using ZFN-based nicking enzyme. Genome Res 7: 1316-1326.