In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

Nature

Volumn 540, Issue 7631, 2016, Pages 144-149

  Suzuki, Keiichiro ^a   Tsunekawa, Yuji ^b   Hernandez Benitez, Reyna ^a,c   Wu, Jun ^a,d   Zhu, Jie ^e,f   Kim, Euiseok J ^a   Hatanaka, Fumiyuki ^a   Yamamoto, Mako ^a   Araoka, Toshikazu ^a,d   Li, Zhe ^g   Kurita, Masakazu ^a   Hishida, Tomoaki ^a   Li, Mo ^a   Aizawa, Emi ^a   Guo, Shicheng ^g   Chen, Song ^g   Goebl, April ^a   Soligalla, Rupa Devi ^a   Qu, Jing ^h,i   Jiang, Tingshuai ^f,j   Fu, Xin ^e,f   Jafari, Maryam ^f   Esteban, Concepcion Rodriguez ^a   Berggren, W Travis ^a   Lajara, Jeronimo ^d   Nuñez Delicado, Estrella ^d   Guillen, Pedro ^d,k   Campistol, Josep M ^l   Matsuzaki, Fumio ^b   Liu, Guang Hui ^i,m,n,o   Magistretti, Pierre ^c   Zhang, Kun ^g   Callaway, Edward M ^a   Zhang, Kang ^e,f,p,q   Belmonte, Juan Carlos Izpisua ^a   more..

^a SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^b RIKEN Center for Biosystems Dynamics Research   (Japan)

^c KING ABDULLAH UNIVERSITY OF SCIENCE AND TECHNOLOGY   (Saudi Arabia)

^d CATHOLIC UNIVERSITY OF MURCIA   (Spain)

^e GUANGZHOU MEDICAL UNIVERSITY   (China)

^f UNIVERSITY OF CALIFORNIA SAN DIEGO   (United States)

^g UNIVERSITY OF CALIFORNIA SAN DIEGO   (United States)

^h INSTITUTE OF ZOOLOGY   (China)

ⁱ UNIVERSITY OF CHINESE ACADEMY OF SCIENCES   (China)

^j Guangzhou KangRui Biological Pharmaceutical Technology Company Ltd   (China)

^k Clinica Centro Fundación Pedro Guillén   (Spain)

^l UNIVERSITY OF BARCELONA   (Spain)

^m INSTITUTE OF BIOPHYSICS   (China)

ⁿ JINAN UNIVERSITY   (China)

^o BEIJING INSTITUTE FOR BRAIN DISORDERS   (China)

^p SICHUAN UNIVERSITY   (China)

^q VA SAN DIEGO HEALTHCARE SYSTEM   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

DNA;

BIOENGINEERING; CELLS AND CELL COMPONENTS; GENOME; HOMOLOGY; NEUROLOGY; NUMERICAL MODEL; RODENT;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; CRISPR CAS SYSTEM; CRISPR CAS9 SYSTEM; DNA INTEGRATION; EMBRYO; HEK293 CELL LINE; HOMOLOGY INDEPENDENT TARGETED INTEGRATION; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO GENE TRANSFER; IN VIVO STUDY; MOUSE; NONHUMAN; PRIORITY JOURNAL; RAT; RETINITIS PIGMENTOSA; VISION; ANIMAL; CELL DIVISION; CYTOLOGY; DISEASE MODEL; GENE EDITING; GENE TARGETING; GENE THERAPY; GENETICS; GENOME; METABOLISM; NERVE CELL; PROCEDURES; SEQUENCE HOMOLOGY;

MAMMALIA; RATTUS;

ANIMALS; CELL DIVISION; CRISPR-CAS SYSTEMS; DISEASE MODELS, ANIMAL; GENE EDITING; GENE KNOCK-IN TECHNIQUES; GENE TARGETING; GENETIC THERAPY; GENOME; NEURONS; RATS; RETINITIS PIGMENTOSA; SEQUENCE HOMOLOGY;

EID: 85000819280     PISSN: 00280836     EISSN: 14764687     Source Type: Journal    
DOI: 10.1038/nature20565     Document Type: Article

References (29)

1. Naldini, L. Gene therapy returns to centre stage. Nature 526, 351-360 (2015).
2. Cox, D. B. T., Platt, R. J. & Zhang, F. Therapeutic genome editing: prospects and challenges. Nat. Med. 21, 121-131 (2015).
3. Mali, P. et al. RNA-guided human genome engineering via Cas9. Science 339, 823-826 (2013).
4. Cong, L. et al. Multiplex genome engineering using CRISPR/Cas systems. Science 339, 819-823 (2013).
5. Lombardo, A. et al. Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat. Methods 8, 861-869 (2011).
6. Genovese, P. et al. Targeted genome editing in human repopulating haematopoietic stem cells. Nature 510, 235-240 (2014).
7. Luchetti, A., Malgieri, A. & Sangiuolo, F. Small fragment homologous replacement (SFHR): sequence-specific modification of genomic DNA in eukaryotic cells by small DNA fragments. Methods Mol. Biol. 1114, 85-101 (2014).
8. Orthwein, A. et al. A mechanism for the suppression of homologous recombination in G1 cells. Nature 528, 422-426 (2015).
9. Lieber, M. R. The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway. Annu. Rev. Biochem. 79, 181-211 (2010).
10. Bétermier, M., Bertrand, P. & Lopez, B. S. Is non-homologous end-joining really an inherently error-prone process? PLoS Genet. 10, e1004086 (2014).
11. Maresca, M., Lin, V. G., Guo, N. & Yang, Y. Obligate ligation-gated recombination (ObLiGaRe): custom-designed nuclease-mediated targeted integration through nonhomologous end joining. Genome Res. 23, 539-546 (2013).
12. Auer, T. O., Duroure, K., De Cian, A., Concordet, J.-P. & Del Bene, F. Highly efficient CRISPR/Cas9-mediated knock-in in zebrafish by homologyindependent DNA repair. Genome Res. 24, 142-153 (2014).
13. Nakade, S. et al. Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9. Nat. Commun. 5, 5560 (2014).
14. Chen, Z.-Y. et al. Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver. Mol. Ther. 3, 403-410 (2001).
15. Chen, Z.-Y., He, C.-Y., Ehrhardt, A. & Kay, M. A. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol. Ther. 8, 495-500 (2003).
16. Wu, J., Corbett, A. H. & Berland, K. M. The intracellular mobility of nuclear import receptors and NLS cargoes. Biophys. J. 96, 3840-3849 (2009).
17. Kameda, Y., Kameya, T. & Frankfurter, A. Immunohistochemical localization of a neuron-specific β-tubulin isotype in the developing chicken ultimobranchial glands. Brain Res. 628, 121-127 (1993).
18. Tabata, H. & Nakajima, K. Efficient in utero gene transfer system to the developing mouse brain using electroporation: visualization of neuronal migration in the developing cortex. Neuroscience 103, 865-872 (2001).
19. Mizutani, K. & Saito, T. Progenitors resume generating neurons after temporary inhibition of neurogenesis by Notch activation in the mammalian cerebral cortex. Development 132, 1295-1304 (2005).
20. Madisen, L. et al. A robust and high-throughput Cre reporting and characterization system for the whole mouse brain. Nat. Neurosci. 13, 133-140 (2010).
21. Samulski, R. J. & Muzyczka, N. AAV-mediated gene therapy for research and therapeutic purposes. Annu. Rev. Virol. 1, 427-451 (2014).
22. Bainbridge, J. W. B. et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med. 358, 2231-2239 (2008).
23. Maguire, A. M. et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248 (2008).
24. Nathwani, A. C. et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N. Engl. J. Med. 371, 1994-2004 (2014).
25. Zincarelli, C., Soltys, S., Rengo, G. & Rabinowitz, J. E. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol. Ther. 16, 1073-1080 (2008).
26. Luo, J. et al. Human retinal progenitor cell transplantation preserves vision. J. Biol. Chem. 289, 6362-6371 (2014).
27. D'Cruz, P. M. et al. Mutation of the receptor tyrosine kinase gene Mertk in the retinal dystrophic RCS rat. Hum. Mol. Genet. 9, 645-651 (2000).
28. Boyden, E. S., Zhang, F., Bamberg, E., Nagel, G. & Deisseroth, K. Millisecondtimescale, genetically targeted optical control of neural activity. Nat. Neurosci. 8, 1263-1268 (2005).
29. Izpisua Belmonte, J. C. et al. Brains, genes, and primates. Neuron 86, 617-631 (2015).