CRISPR-Cas Technology, the Tool of the Future
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The CRISPR-Cas system discovered in the eighties consists of a series of RNA proteins that signal and messengers of the immune system in prokaryotic organisms to protect against invading antigens of various sources. After the CRISPR-Cas discovery, many research advances were made in the following decades regarding knowledge, techniques, and applications. This showed the system could edit the DNA in an exact and specific manner, which made it very promising to exploit it in various fields, such as the therapeutic field. New therapies of various diseases, industrial applications, food manufacturing, among others, make its impact quite relevant. The following review will focus on the fundamental understanding and implications of CRISPR-Cas techniques has with an ethical and legal view. In addition, to explore some of the applications present and future in the healthcare department, some methods of drug delivery in gene therapy, and new research that is being developed with the CRISPR-Cas technology.
References
-
Abdelaal, A. S., & Yazdani, S. S. (2020). Development and use of CRISPR in industrial applications. En Genome Engineering via CRISPR-Cas9 System. 177-197.
Google Scholar
1
-
Alkhnbashi, O. S., Meier, T., Mitrofanov, A., Backofen, R., & Voß, B. (2020). CRISPR-Cas bioinformatics. Methods, 172, 3-11.
Google Scholar
2
-
Alsaiari, S. K., Patil, S., Alyami, M., Alamoudi, K. O., Aleisa, F. A., Merzaban, J. S., et al. (2018). Endosomal Escape and Delivery of CRISPR/Cas9 Genome Editing Machinery Enabled by Nanoscale Zeolitic Imidazolate Framework. Journal of the American Chemical Society, 140(1), 143-146.
Google Scholar
3
-
Araldi, R. P., Khalil, C., Grignet, P. H., Teixeira, M. R., de Melo, T. C., Módolo, D. G., et al. (2020). Medical applications of clustered regularly interspaced short palindromic repeats (CRISPR/Cas) tool: A comprehensive overview. Gene, 745, 144636.
Google Scholar
4
-
Arias-Fuenzalida, J., Jarazo, J., Qing, X., Walter, J., Gomez-Giro, G., Nickels, S. L., et al. (2017). FACS-Assisted CRISPR-Cas9 Genome Editing Facilitates Parkinson’s Disease Modeling. Stem Cell Reports, 9(5), 1423-1431.
Google Scholar
5
-
Banaszynski, L. A., Chen, L.-C., Maynard-Smith, L. A., Ooi, A. G. L., & Wandless, T. J. (2006). A rapid, reversible, and tunable method to regulate protein function in living cells using synthetic small molecules. Cell, 126(5), 995-1004.
Google Scholar
6
-
Barajas, A. & Hernández, M. (2020). La bioética del sistema CRISPR-Cas9 como terapia génica en enfermedades de importancia mundial. Ciencia Huasteca Boletín Científico de la Escuela Superior de Huejutla, 8(16), 29-33.
Google Scholar
7
-
Bonini, A., Poma, N., Vivaldi, F., Kirchhain, A., Salvo, P., Bottai, D., et al. (2021). Advances in biosensing: The CRISPR/Cas system as a new powerful tool for the detection of nucleic acids. Journal of Pharmaceutical and Biomedical Analysis, 192, 113645.
Google Scholar
8
-
Capella, V. B. (2016). La revolución de la edición genética mediante CRISPR-Cas9 y los desafíos éticos y regulatorios que comporta. Cuadernos de Bioética, 19(2), 223-239.
Google Scholar
9
-
Cárdenas, R. (2019). El Derecho ante la técnica de edición genética CRISPR. Acta bioethica, 25(2), 187-197.
Google Scholar
10
-
Chávez, V. M. (2018). El sistema de edición genética CRISPR/Cas y su uso como antimicrobiano específico. Revista especializada en ciencias químico-biológicas, 21(2), 8. (Spanish).
Google Scholar
11
-
Cho, S., Shin, J., & Cho, B.-K. (2018). Applications of CRISPR/Cas System to Bacterial Metabolic Engineering. International Journal of Molecular Sciences, 19(4), 1089.
Google Scholar
12
-
Cong, L., Cox, D. B. T., Heidenreich, M., Platt, R. J., SWIECH, L., & Zhang, F. (2016). Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing. European Union Patent N.o EP3079725A1. Available from: https://patents.google.com/patent/EP3079725A1/en?oq=crispr+cas
Google Scholar
13
-
Cota J, Sandoval S, Gaytan Y, Diaz N, Vega B, Padilla E, Díaz N.E. (2020). Nuevos modelos transgénicos para el estudio de la enfermedad de Parkinson basados en sistemas de edición con nucleasas. Neurología; 35(7), 486-499. (Spanish).
Google Scholar
14
-
D’Astolfo, D. S., Pagliero, R. J., Pras, A., Karthaus, W. R., Clevers, H., Prasad, V., et al. (2015). Efficient intracellular delivery of native proteins. Cell, 161(3), 674-690.
Google Scholar
15
-
De Buhr, H., & Lebbink, R. J. (2018). Harnessing CRISPR to combat human viral infections. Current Opinion in Immunology, 54, 123-129.
Google Scholar
16
-
Dominguez, A. A., Lim, W. A., & Qi, L. S. (2016). Beyond editing: Repurposing CRISPR-Cas9 for precision genome regulation and interrogation. Nature Reviews. Molecular Cell Biology, 17(1), 5-15.
Google Scholar
17
-
Doudna, J. A. & Charpentier, E. (2014) The new frontier of genome engineering with CRISPR-Cas9. Science, 346(6213).
Google Scholar
18
-
Ekman F, Ojala D, Adil M, Lopez P, Schaffer D, Gaj T. (2019) CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in Huntington's Disease Mouse Model. Molecular Therapy - Nucleic Acids, 17;829-839.
Google Scholar
19
-
Enzmann, B. (2019). How CRISPR Is Accelerating Drug Discovery. GEN - Genetic Engineering and Biotechnology News, 39(1).
Google Scholar
20
-
Fellmann, C., Gowen, B. G., Lin, P.-C., Doudna, J. A., & Corn, J. E. (2017). Cornerstones of CRISPR-Cas in drug discovery and therapy. Nature Reviews. Drug Discovery, 16(2), 89-100.
Google Scholar
21
-
Finn, J. D., Smith, A. R., Patel, M. C., Shaw, L., Youniss, M. R., van Heteren, J., et al. (2018). A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing. Cell Reports, 22(9), 2227-2235.
Google Scholar
22
-
Foss, D. V., Hochstrasser, M. L., & Wilson, R. C. (2019). Clinical applications of CRISPR-based genome editing and diagnostics. Transfusion, 59(4), 1389-1399.
Google Scholar
23
-
Freiermuth, J. L., Powell-Castilla, I. J., & Gallicano, G. I. (2018). Toward a CRISPR Picture: Use of CRISPR/Cas9 to Model Diseases in Human Stem Cells In Vitro. Journal of Cellular Biochemistry, 119(1), 62-68.
Google Scholar
24
-
García, K., Lehka, B. J., & Mortensen, U. H. (2017). SWITCH: A dynamic CRISPR tool for genome engineering and metabolic pathway control for cell factory construction in Saccharomyces cerevisiae. Microbial Cell Factories, 16(1), 25.
Google Scholar
25
-
Gutiérrez, D. & Salazar, L. (2017) Tema 2017: CRISPR-Cas: Utilidad clínica de la edición genómica como opción terapéutica. Revista Clínica de la Escuela de Medicina UCR-HSJD, 7(6). (Spanish).
Google Scholar
26
-
Horvath, P., & Barrangou, R. (2010). CRISPR/Cas, the Immune System of Bacteria and Archaea. Science, 327(5962), 167-170.
Google Scholar
27
-
Hoy, M. A. (2019). CRISPR-Cas Genome Editing: Another Revolution in Molecular Biology. En Insect Molecular Genetics, 345-361.
Google Scholar
28
-
Hu, X. (2016). CRISPR/Cas9 system and its applications in human hematopoietic cells. Blood Cells, Molecules, and Diseases, 62, 6-12.
Google Scholar
29
-
Jaitin, D. A., Weiner, A., Yofe, I., Lara-Astiaso, D., Keren-Shaul, H., David, E., et al. (2016). Dissecting Immune Circuits by Linking CRISPR-Pooled Screens with Single-Cell RNA-Seq. Cell, 167(7), 1883-1896.e15.
Google Scholar
30
-
Jiang, C., Lin, X., & Zhao, Z. (2019). Applications of CRISPR/Cas9 Technology in the Treatment of Lung Cancer. Trends in Molecular Medicine, 25(11), 1039-1049.
Google Scholar
31
-
Kang, H., Minder, P., Park, M. A., Mesquitta, W.-T., Torbett, B. E., & Slukvin, I. I. (2015). CCR5 Disruption in Induced Pluripotent Stem Cells Using CRISPR/Cas9 Provides Selective Resistance of Immune Cells to CCR5-tropic HIV-1 Virus. Molecular Therapy - Nucleic Acids, 4: e268.
Google Scholar
32
-
Khan, S. H. (2019). Genome-Editing Technologies: Concept, Pros, and Cons of Various Genome-Editing Techniques and Bioethical Concerns for Clinical Application. Molecular Therapy - Nucleic Acids, 16, 326-334.
Google Scholar
33
-
Klompe, S. E., & Sternberg, S. H. (2020). CRISPR–Cas immune systems and genome engineering. Rosenberg’s Molecular and Genetic Basis of Neurological and Psychiatric Disease, 157-177.
Google Scholar
34
-
Lammoglia, M. F., Lozano, R., García, C. D., Avilez, C. M., Trejo, V., Muñoz, R. B., et al. (2016). La revolución en ingeniería genética: Sistema CRISPR/Cas. 13.
Google Scholar
35
-
Lee, J., Bayarsaikhan, D., Arivazhagan, R., Park, H., Lim, B., Gwak, P., Lee, B. (2019). CRISPR-Cas9 Edited sRAGE-MSCs Protect Neuronal Death in Parkinson’s Disease Model. International Journal of Stem Cells, 12(1), 114-24.
Google Scholar
36
-
Luo, J., Padhi, P., Jin, H., Anantharam, V., Zenitsky, G., Wang, Q., Kanthasamy, A. G. (2019). Utilization of the CRISPR-Cas9 Gene Editing System to Dissect Neuroinflammatory and Neuropharmacological Mechanisms in Parkinson’s Disease. Journal of Neuroimmune Pharmacology, 14(4), 595-607.
Google Scholar
37
-
Ortiz-Virumbrales M, Moreno C, Kruglikov I, Marazuela P, Sproul A, Jacob S., et al. (2017) CRISPR/Cas9-Correctable mutation-related molecular and physiological phenotypes in iPSC-derived Alzheimer’s PSEN2 N141I neurons. Acta Neuropathologica Communications, 5(1).
Google Scholar
38
-
Pahan K. (2019). A Broad Application of CRISPR Cas9 in Infectious, Inflammatory and Neurodegenerative Diseases. Journal of Neuroimmune Pharmacology, 14(4), 534–6.
Google Scholar
39
-
Patel, S., Ashwanikumar, N., Robinson, E., DuRoss, A., Sun, C., Murphy-Benenato, K. E., et al. (2017). Boosting Intracellular Delivery of Lipid Nanoparticle-Encapsulated mRNA. Nano Letters, 17(9), 5711-5718.
Google Scholar
40
-
Quílez, Á. (2017). Aplicaciones de CRISPR en la generación y síntesis de nuevos fármacos. MoleQla: revista de Ciencias de la Universidad Pablo de Olavide, 27, 10. (Spanish).
Google Scholar
41
-
Raschmanová, H., Weninger, A., Glieder, A., Kovar, K., & Vogl, T. (2018). Implementing CRISPR-Cas technologies in conventional and non-conventional yeasts: Current state and future prospects. Biotechnology Advances, 36(3), 641-665.
Google Scholar
42
-
Ruiz; OH. (2018). El sistema CRISPR-Cas y su aplicación en las enfermedades infecciosas. Hechos Microbiológicos, 9(1-2), 9-11.
Google Scholar
43
-
Sapranauskas, R., Gasiunas, G., Fremaux, C., Barrangou, R., Horvath, P., & Siksnys, V. (2011). The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli. Nucleic Acids Research, 39(21), 9275-9282.
Google Scholar
44
-
Scott, A. (2018). A CRISPR path to drug discovery. Nature, 555.
Google Scholar
45
-
Senturk, S., Shirole, N. H., Nowak, D. G., Corbo, V., Pal, D., Vaughan, A., Tuveson, D. A., Trotman, L. C., Kinney, J. B., & Sordella, R. (2017). Rapid and tunable method to temporally control gene editing based on conditional Cas9 stabilization. Nature Communications, 8, 14370.
Google Scholar
46
-
Shanmugam, S., Ngo, H.-H., & Wu, Y.-R. (2020). Advanced CRISPR/Cas-based genome editing tools for microbial biofuels production: A review. Renewable Energy, 149(C), 1107-1119.
Google Scholar
47
-
Shin, J., Lee, N., Cho, S., & Cho, B.-K. (2018). Targeted Genome Editing Using DNA-Free RNA-Guided Cas9 Ribonucleoprotein for CHO Cell Engineering. Methods in Molecular Biology (Clifton, N.J.), 1772, 151-169.
Google Scholar
48
-
Shipman, S. L., Nivala, J., Macklis, J. D., & Church, G. M. (2017). CRISPR–Cas encoding of a digital movie into the genomes of a population of living bacteria. Nature, 547(7663), 345-349.
Google Scholar
49
-
Singh, V. (2020). An introduction to genome editing CRISPR-Cas systems. Genome Engineering via CRISPR-Cas9 System, 1-13.
Google Scholar
50
-
Smalley, E. (2016). CRISPR mouse model boom, rat model renaissance. Nature Biotechnology, 34(9), 893-894.
Google Scholar
51
-
Song, H.-Y., Chiang, H.-C., Tseng, W.-L., Wu, P., Chien, C.-S., Leu, H.-B., et al. (2016). Using CRISPR/Cas9-Mediated GLA Gene Knockout as an In Vitro Drug Screening Model for Fabry Disease. International Journal of Molecular Sciences, 17(12), 2089.
Google Scholar
52
-
Soppe, J. A., & Lebbink, R. J. (2017). Antiviral Goes Viral: Harnessing CRISPR/Cas9 to Combat Viruses in Humans. Trends in Microbiology, 25(10), 833-850.
Google Scholar
53
-
Tu, Z., Yang, W., Yan, S., Guo, X., & Li, X.-J. (2015). CRISPR/Cas9: A powerful genetic engineering tool for establishing large animal models of neurodegenerative diseases. Molecular Neurodegeneration, 10(1), 35.
Google Scholar
54
-
Wang, D., Mou, H., Li, S., Li, Y., Hough, S., Tran, K., et al. (2015). Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses. Human Gene Therapy, 26(7), 432-442.
Google Scholar
55
-
Wilbie, D., Walther, J., & Mastrobattista, E. (2019). Delivery Aspects of CRISPR/Cas for in Vivo Genome Editing. Accounts of Chemical Research, 52(6), 1555-1564.
Google Scholar
56
-
Xu, W. (2019). Microinjection and Micromanipulation: A Historical Perspective. Methods in Molecular Biology (Clifton, N.J.), 1874, 1-16.
Google Scholar
57
-
Xue, W., Chen, S., Yin, H., Tammela, T., Papagiannakopoulos, T., Joshi, N. S., et al. (2014). CRISPR-mediated direct mutation of cancer genes in the mouse liver. Nature, 514(7522), 380-384.
Google Scholar
58
-
Yao, R., Liu, D., Jia, X., Zheng, Y., Liu, W., & Xiao, Y. (2018). CRISPR-Cas9/Cas12a biotechnology and application in bacteria. Synthetic and Systems Biotechnology, 3(3), 135-149.
Google Scholar
59
-
Yen, J., Fiorino, M., Liu, Y., Paula, S., Clarkson, S., Quinn, L., et al. 2018). TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells. Scientific Reports, 8(1), 16304.
Google Scholar
60
-
Yin, H., Song, C.-Q., Dorkin, J. R., Zhu, L. J., Li, Y., Wu, Q., et al. (2016). Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo. Nature Biotechnology, 34(3), 328-333.
Google Scholar
61
-
Yu, W., & Wu, Z. (2020). Ocular delivery of CRISPR/Cas genome editing components for treatment of eye diseases. Advanced Drug Delivery Reviews, S0169409X20300570.
Google Scholar
62
-
Zetsche, B., Volz, S. E., & Zhang, F. (2015). A split-Cas9 architecture for inducible genome editing and transcription modulation. Nature Biotechnology, 33(2), 139-142.
Google Scholar
63
-
Zhang, Z., Wan, T., Chen, Y., Chen, Y., Sun, H., et al. (2019). Cationic Polymer-Mediated CRISPR/Cas9 Plasmid Delivery for Genome Editing. Macromolecular Rapid Communications, 40(5), e1800068.
Google Scholar
64
-
Zhang, Z.-Y., Thrasher, A. J., & Zhang, F. (2020). Gene therapy and genome editing for primary immunodeficiency diseases. Genes & Diseases, 7(1), 38-51.
Google Scholar
65
-
Jian, C., Wei, J., & Yue, P. (2016). A CRISPR-Cas9 system targeting apoCIII and its aplication. China Patent N.º CN105462968A. https://patents.google.com/patent/CN105462968A/zh?oq=crispr+cas9
Google Scholar
66
-
Zhang, J. & Qin X. (2016). Target sequence and sgRNA of human CCR5 gene recognized by CRISPR-Cas9 system in Neisseria meningitidis and its application. China Patent N.º CN105331609A. https://patents.google.com/patent/CN105331609A/en?oq=crispr+cas9
Google Scholar
67
-
Cai, Z., Mou, L., Chen, P., Xie, C., Zhang, J., Gao, H. et al, (2016). CRISPR-Cas9 specific knockout method of pig SLA-1 gene and sgRNA for specific targeting of SLA-1 gene. World Intellectual Property Organization Patent N.º WO2016197359A1. https://patents.google.com/patent/WO2016197359A1/en?oq=crispr+cas9
Google Scholar
68