CRISPR/Cas9: Implications for modeling and therapy of neurodegenerative diseases

Frontiers in Molecular Neuroscience

Volumn 9, Issue APR, 2016, Pages

  Yang, Weili ^a   Tu, Zhuchi ^a   Sun, Qiang ^a   Li, Xiao Jiang ^a,b  

^a INSTITUTE OF GENETICS AND DEVELOPMENTAL BIOLOGY   (China)

^b EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Animal models; CRISPR Cas9; Neurodegenerative diseases

Indexed keywords

ALPHA SYNUCLEIN; COMPLEMENTARY RNA; CRISPR ASSOCIATED PROTEIN; CRISPR ASSOCIATED PROTEIN 9; DYSTROPHIN; GUIDE RNA; HUNTINGTIN; NUCLEASE; PARKIN; UNCLASSIFIED DRUG;

ALLELE; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; DEGENERATIVE DISEASE; DNA BINDING; DNA CLEAVAGE; EPIGENETICS; GENE DELETION; GENE FUNCTION; GENE MUTATION; GENE TARGETING; HUMAN; HUNTINGTON CHOREA; MOSAICISM; MUSCULAR DYSTROPHY; NONHUMAN; PARKINSON DISEASE; REVIEW; ZYGOTE;

EID: 84971330670     PISSN: 16625099     EISSN: None     Source Type: Journal    
DOI: 10.3389/fnmol.2016.00030     Document Type: Review

References (41)

1. Barrangou, R., Fremaux, C., Deveau, H., Richards, M., Boyaval, P., Moineau, S., et al. (2007). CRISPR provides acquired resistance against viruses in prokaryotes. Science 315, 1709-1712. doi: 10.1126/science.1138140
2. Brouns, S. J. J., Jore, M. M., Lundgren, M., Westra, E. R., Slijkhuis, R. J. H., Snijders, A. P. L., et al. (2008). Small CRISPR RNAs guide antiviral defense in prokaryotes. Science 321, 960-964. doi: 10.1126/science. 1159689
3. Callaway, E. (2016). UK scientists gain licence to edit genes in human embryos. Nature 530: 18. doi: 10.1038/nature.2016.19270
4. Chen, B. H., Gilbert, L. A., Cimini, B. A., Schnitzbauer, J., Zhang, W., Li, G. W., et al. (2014). Dynamic imaging of genomic loci in living human cells by an optimized CRISPR/cas system. Cell 155, 1479-1491. doi: 10.1016/j.cell.2013. 12.001
5. Chen, Y., Zheng, Y., Kang, Y., Yang, W., Niu, Y., Guo, X., et al. (2015). Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9. Hum. Mol. Genet. 24, 3764-3774. doi: 10.1093/hmg/ddv120
6. Chu, V. T., Weber, T., Wefers, B., Wurst, W., Sander, S., Rajewsky, K., et al. (2015). Increasing the efficiency of homology-directed repair for CRISPRCas9- induced precise gene editing in mammalian cells. Nat. Biotechnol. 33, 543-548. doi: 10.1038/nbt.3198
7. Daley, J. M., and Sung, P. (2014). 53BP1, BRCA1 and the choice between recombination and end joining at DNA double-strand breaks. Mol. Cell. Biol. 34, 1380-1388. doi: 10.1128/MCB.01639-13
8. Fu, Y., Sander, J. D., Reyon, D., Cascio, V. M., and Joung, J. K. (2014). Improving CRISPR-Cas nuclease specificity using truncated guide RNAs. Nat. Biotechnol. 32, 279-284. doi: 10.1038/nbt.2808
9. Heidenreich, M., and Zhang, F. (2016). Applications of CRISPR-Cas systems in neuroscience. Nat. Rev. Neurosci. 17, 36-44. doi: 10.1038/nrn.2015.2
10. Heyer, W. D., Ehmsen, K. T., and Liu, J. (2010). Regulation of homologous recombination in eukaryotes. Annu. Rev. Genet. 44, 113-139. doi: 10. 1146/annurev-genet-051710-150955
11. Hsu, P. D., Lander, E. S., and Zhang, F. (2014). Development and applications of CRISPR-Cas9 for genome engineering. Cell 157, 1262-1278. doi: 10.1016/j.cell. 2014.05.010
12. Hsu, P. D., Scott, D. A., Weinstein, J. A., Ran, F. A., Konermann, S., Agarwala, V., et al. (2013). DNA targeting specificity of RNA-guided Cas9 nucleases. Nat. Biotechnol. 31, 827-832. doi: 10.1038/nbt.2647
13. Incontro, S., Asensio, C. S., Edwards, R. H., and Nicoll, R. A. (2014). Efficient, complete deletion of synaptic proteins using CRISPR. Neuron 83, 1051-1057. doi: 10.1016/j.neuron.2014.07.043
14. Ishino, Y., Shinagawa, H., Makino, K., Amemura, M., and Nakata, A. (1987). Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli and identification of the gene product. J. Bacteriol. 169, 5429-5433.
15. Jansen, R., van Embden, J. D. A., Gaastra, W., and Schouls, L. M. (2002). Identification of genes that are associated with DNA repeats in prokaryotes. Mol. Microbiol. 43, 1565-1575. doi: 10.1046/j.1365-2958.2002. 02839.x
16. Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., and Charpentier, E. (2012). A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337, 816-821. doi: 10.1126/science.1225829
17. Karginov, F. V., and Hannon, G. J. (2010). The CRISPR system: small RNA-guided defense in bacteria and archaea. Mol. Cell 37, 7-19. doi: 10.1016/j.molcel.2009. 12.033
18. Kim, S., Kim, D., Cho, S. W., Kim, J., and Kim, J. S. (2014). Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res. 24, 1012-1019. doi: 10.1101/gr. 171322.113
19. Koike-Yusa, H., Li, Y. L., Tan, E. P., Velasco-Herrera, M. D., and Yusa, K. (2014). Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library. Nat. Biotechnol. 32, 267-273. doi: 10.1038/nbt. 2800
20. Larson, M. H., Gilbert, L. A., Wang, X. W., Lim, W. A., Weissman, J. S., and Qi, L. S. (2013). CRISPR interference (CRISPRi) for sequence-specific control of gene expression. Nat. Protoc. 8, 2180-2196. doi: 10.1038/nprot.2013.132
21. Li, X. J., and Li, S. H. (2011). Proteasomal dysfunction in aging and Huntington disease. Neurobiol. Dis. 43, 4-8. doi: 10.1016/j.nbd.2010.11.018
22. Liang, P., Xu, Y., Zhang, X., Ding, C., Huang, R., Zhang, Z., et al. (2015). CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein Cell 6, 363-372. doi: 10.1007/s13238-015-0153-5
23. Long, C., Amoasii, L., Mireault, A. A., McAnally, J. R., Li, H., Sanchez-Ortiz, E., et al. (2016). Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351, 400-403. doi: 10. 1126/science.aad5725
24. Mali, P., Esvelt, K. M., and Church, G. M. (2013). Cas9 as a versatile tool for engineering biology. Nat. Methods 10, 957-963. doi: 10.1038/nmeth.2649
25. Maruyama, T., Dougan, S. K., Truttmann, M. C., Bilate, A. M., Ingram, J. R., and Ploegh, H. L. (2015). Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining. Nat. Biotechnol. 33, 538-542. doi: 10.1038/nbt.3190
26. Mojica, F. J., Díez-Villaseñor, C., Soria, E., and Juez, G. (2000). Biological significance of a family of regularly spaced repeats in the genomes of Archaea, Bacteria and mitochondria. Mol. Microbiol. 36, 244-246. doi: 10.1046/j.1365-2958.2000.01838.x
27. Nelson, C. E., Hakim, C. H., Ousterout, D. G., Thakore, P. I., Moreb, E. A., Castellanos Rivera, R. M., et al. (2016). In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 351, 403-407. doi: 10.1126/science.aad5143
28. Niu, Y., Shen, B., Cui, Y., Chen, Y., Wang, J., Wang, L., et al. (2014). Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos. Cell 156, 836-843. doi: 10.1016/j.cell.2014. 01.027
29. Platt, R. J., Chen, S. D., Zhou, Y., Yim, M. J., Swiech, L., Kempton, H. R., et al. (2014). CRISPR-Cas9 knockin mice for genome editing and cancer modeling. Cell 159, 440-455. doi: 10.1016/j.cell.2014.09.014
30. Qi, L. S., Larson, M. H., Gilbert, L. A., Doudna, J. A., Weissman, J. S., Arkin, A. P., et al. (2013). Repurposing CRISPR as an RNA-guided platform for sequencespecific control of gene expression. Cell 152, 1173-1183. doi: 10.1016/j.cell. 2013.02.022
31. Sander, J. D., and Joung, J. K. (2014). CRISPR-Cas systems for editing, regulating and targeting genomes. Nat. Biotechnol. 32, 347-355. doi: 10.1038/nbt.2842
32. Shalem, O., Sanjana, N. E., Hartenian, E., Shi, X., Scott, D. A., Mikkelsen, T. S., et al. (2014). Genome-scale CRISPR-Cas9 knockout screening in human cells. Science 343, 84-87. doi: 10.1126/science.1247005
33. Straub, C., Granger, A. J., Saulnier, J. L., and Sabatini, B. L. (2014). CRISPR/Cas9- mediated gene knock-down in post-mitotic neurons. PLoS One 9: e105584. doi: 10.1371/journal.pone.0105584
34. Sung, Y. H., Kim, J. M., Kim, H. T., Lee, J., Jeon, J., Jin, Y., et al. (2014). Highly efficient gene knockout in mice and zebrafish with RNA-guided endonucleases. Genome Res. 24, 125-131. doi: 10.1101/gr.163394.113
35. Swiech, L., Heidenreich, M., Banerjee, A., Habib, N., Li, Y., Trombetta, J., et al. (2015). In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9. Nat. Biotechnol. 33, 102-106. doi: 10.1038/nbt.3055
36. Tabebordbar, M., Zhu, K., Cheng, J. K., Chew, W. L., Widrick, J. J., Yan, W. X., et al. (2016). In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351, 407-411. doi: 10.1126/science.aad5177
37. Walters, B. J., Azam, A. B., Gillon, C. J., Josselyn, S. A., and Zovkic, I. B. (2016). Advanced in vivo use of CRISPR/Cas9 and anti-sense DNA inhibition for gene manipulation in the brain. Front. Genet. 6: 362. doi: 10.3389/fgene.2015.00362
38. Wang, X., Cao, C., Huang, J., Yao, J., Hai, T., Zheng, Q., et al. (2016). One-step generation of triple gene-targeted pigs using CRISPR/Cas9 system. Sci. Rep. 6: 20620. doi: 10.1038/srep20620
39. Wu, Y., Liang, D., Wang, Y., Bai, M., Tang, W., Bao, S., et al. (2013). Correction of a genetic disease in mouse via use of CRISPR-Cas9. Cell Stem Cell 13, 659-662. doi: 10.1016/j.stem.2013.10.016
40. Zhou, X., Xin, J., Fan, N., Zou, Q., Huang, J., Ouyang, Z., et al. (2015). Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer. Cell. Mol. Life Sci. 72, 1175-1184. doi: 10.1007/s00018-014-1744-7
41. Zhou, Y. X., Zhu, S. Y., Cai, C. Z., Yuan, P. F., Li, C. M., Huang, Y. Y., et al. (2014). High-throughput screening of a CRISPR/Cas9 library for functional genomics in human cells. Nature 509, 487-491. doi: 10.1038/nature13166