Clustered Regularly Interspaced Short Palindromic Repeats- a new era in Genomic technology and its applications in Theranostics
DOI:
https://doi.org/10.55766/sujst-2023-04-e0229Keywords:
CRISPR Genomic editing, theranostics, therapeuticsAbstract
Clustered Regularly Interspaced Short Palindromic Repeats is termed as CRISPR. Bacteria having "spacer" sequences of deoxyribonucleic acid between the repeats of genomic sequences that resemble genomic sequences in viruses were found to have repetitive DNA sequences known as CRISPR.The CRISPR technology in genetic engineering has revolutionalized the field of medicine in treating various genetically linked diseases that are difficult to treat such as Lebers Congenital amaurosis, in which there is bi-allelic deletion caused by mutation in mitochondrial DNA MT-ND4(NADH ubiquinone oxidoreductase chain4) 1178G are treated by intravitreous injection of AAV2-ND4 produced by CRISPR-Cas9 genomic editing technology.CRISPR-Cas9 genomic editing is used to remove the expression of receptor Enhancer Protein-6 (Reep6 p.Leu135Pro ) gene that causes retinitis pigmentosa. Open angle Glaucoma caused by mutations in myocilin (MYOR) gene was effectively removed out by CRISPR-Cas9 editing technology. Mutation in KRT-12 gene that caused Meesman epithelial corneal dystrophy ( MECD) was alleviated by Cas9/sgRNA injection into the stroma of cornea. CRISPR-Cas9 genomic sequencing used in the treatment of Haemophilia -B , an inherited disease caused by mutation of factor IX gene Y371D was successfully modified by CRISPR-
References
Ahmad, I. (2022). CRISPR/Cas9-a promising therapeutic tool to cure blindness: Current scenario and future prospects. International Journal of Molecular Sciences, 23(19): 11482. https://doi.org/10.3390/ijms231911482
Baghini, S.S., Gardanova, Z.R., Abadi, S.A.H., Zaman, B.A., İlhan, A., Shomali, N., Adili, A., Moghaddar, R., and Yaseri, A.F. (2022). CRISPR/Cas9 application in cancer therapy: a pioneering genome-editing tool. Cellular & Molecular Biology Letters, 27:35. https://doi.org/10.1186/ s11658-022-00336-6
Behrens-Gawlik, V., Mearini, G., Gedicke-Hornung, C., Richard, P., and Carrier, L. (2014). MYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correction. Pflügers Archiv - European Journal of Physiology, 466:215-223. https://doi.org/10.1007/s00424-013-1409-7
Chadwick, A.C., and Musunuru, K. (2018). CRISPR-Cas9 genome editing for treatment of atherogenic dyslipidemia. Arterioscler. Arteriosclerosis, Thrombosis, and Vascular Biology, 38(1):12-18. https://doi.org/10.1161/ATVBAHA. 117.309326
Cho, E.Y., Ryu, J.-Y., Lee, H.A.R., Hong, S.H., Park, H.S., Hong, K.S., Park, S.-G., Kim, H.P., and Yoon, T.-J. (2019). Lecithin nano-liposomal particle as a CRISPR/Cas9 complex delivery system for treating type 2 diabetes. Journal of Nanobiotechnology, 19:19. https://doi.org/ 10.1186/s12951-019-0452-8
Dash, H.R. and Arora, M. (2022). CRISPR-CasB technology in forensic DNA analysis: challenges and solutions. Applied Microbiology and Biotechnology, 106:4367-4374. https://doi.org/10.1007/s00253-022-12016-8
Dukhovny, A., Lamkiewicz, K., Chen, Q., Fricke, M., Jabrane-Ferrat, N., Marz, M., Jung, J.U., and Sklan, E.H. (2019). A CRISPR activation screen identifies genes that protect against Zika virus infection. Journal of Virology, 93(16):00211-19. https://doi.org/10.1128/JVI.00211-19
Foy, S.P., Jacoby, K., Bota, D.A., Hunter, T., Pan, Z., Stawiski, E., Ma, Y., Lu, W., Peng, S., Wang, C.L., Yuen, B., Dalmas, O., Heeringa, K., Sennino, B., Conroy, A., Bethune, M.T., Mende, I., White, W., Kukreja, M., Gunturu, S., Humphrey, E., Hussaini, A., An, D., Litterman, A.J., Quach, B.B., Ng, A.H.C., Lu, Y., Smith, C., Campbell, K.M., Anaya, D., Skrdlant, L., Huang, E.Y., Mendoza, V., Mathur, J., Dengler, L., Purandare, B., Moot, R., Yi, M.C., Funke, R., Sibley, A., Stallings-Schmitt, T., Oh, D.Y., Chmielowski, B., Abedi, M., Yuan, Y., Sosman, J.A., Lee, S.M., Schoenfeld, A.J., Baltimore, D., Heath, J.R., Franzusoff, A., Ribas, A., Rao, A.V., and Mandl, S.J. (2023). Non-viral precision T cell receptor replacement for personalized cell therapy. Nature. 615(7953):687-696. https://doi.org/10.1038/ s41586-022-05531-1
Frangoul, H., Altshuler, D., Cappellini, M.D., Chen, Y.-S., Domm, J., Eustace, B.K., Foell, J., de la Fuente, J., Grupp, S., Handgretinger, R., Ho, T.W., Kattamis, A., Kernytsky, A., Lekstrom-Himes, J., Li, A.M., Locatelli, F., Mapara, M.Y., de Montalembert, M., Rondelli, D., Sharma, A., Sheth, S., Soni, S., Steinberg, M.H., Wall, D., Yen, A., and Corbacioglu, S. (2021). CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. The New England Journal of Medicine, 384:252-260. https://doi.org/10.1056/ NEJMoa2031054
Gillmore, J.D., Gane, Ed., Taubel, J., Kao, J., Fontana, M., Maitland, M.L., Seitzer, J., O’Connell, D., Walsh, K.R., Wood, K., Phillips, J., Xu, Y., Amaral, A., Boyd, A.P., Cehelsky, J.E., McKee, M.D., Schiermeier, A., Harari, O., Murphy, A., Kyratsous, C.A., Zambrowicz, B., Soltys, R., Gutstein, D.E., Leonard, J., Sepp-Lorenzino, L., and Lebwohl, D. (2021). CRISPR-Cas9: in vivo gene editing for transthyretin amyloidosis. New England Journal of Medicine, 385:493-502. https://doi.org/10.1056/NEJMoa 2107454
Gong, Tao., Tang, B., Zhou, X., Zeng, J., Lu, M., Guo, X., Peng X., Lei, L., Gong, B., and Li, Y. (2018). Genome Editing in Streptococcus mutans through self-targeting CRISPR arrays. Molecular Oral Microbiology, 33(6):440-449. https://doi.org/10.1111/omi.12247
Graham, C. and Hart, S. (2021). CRISPR/Cas9 gene-editing therapies for cystic fibrosis Expert Opinion on Biological Therapy, 21(6):767-780. https://doi.org/10.1080/14712598. 2021.1869208
Hsu, P.D., Lander, E.S., and Zhang, F. (2014). Development and applications of CRISPR-Cas9 for genome engineering. Cell, 157(6):1262-1278. https://doi.org/10.1016/j.cell.2014. 05.010
Kaneski, C.R., Hanover, J.A., and Hoffman, U.H.S. (2022). Generation of an in vitro model for peripheral neuropathy in Fabry disease using CRISPR-Cas9 in the nociceptive dorsal root ganglion cell ganglion cell line 50B11. Molecular Genetics and Metabolism Reports, 31:100871. https://doi.org/10.1016/j.ymgmr.2022.100871
Kennedy, E.M., Bassit, L.C., Mueller, H., Kornepati, A.V.R., Bogerd, H.P., Nie, T., Chatterjee, P., Javanbakht, H., Schinazi R.F., and Cullen, B.R. (2015). Suppression of hepatitis B virus DNA accumulation in chronically infected cells using a bacterial CRISPR/Cas RNA-guided DNA endonuclease. Virology, 476:196-205. https://doi.org/ 10.1016/j.virol.2014.12.001
Kennedya, E.M., Kornepatia, A.V.R., Goldsteinb, M., Bogerda, H.P., Polinga, B.C., Whisnanta, A.W., Kastanb, M.B., and Cullena, B.R. (2014). Inactivation of the human papillomavirus E6 or E7 gene in cervical carcinoma cells by using a bacterial CRISPR/Cas RNA-guided endonuclease. Journal of Virology, 88(20):11965-11972. https://doi.org/ 10.1128/JVI.01879-14
Lee, M.C.S., Lindner, S.E., Lopez-Rubio, J.-J., and Llinás, M. (2019). Cutting back Malaria: CRISPR/Cas9 genome editing of Plasmodium. Briefings in Functional Genomics, 18(5):281-289. https://doi.org/10.1093/bfgp/elz012
Lee, M.H., Shin, J.I., Yang, J.W., Lee, K.H., Cha, D.H., Hong, J.B., Park, Y., Choi, E., Tizaoui, K., Koyanagi, A., Jacob, L., Park, S., Kim, J.H., and Smith, L. (2022). Genome editing using CRISPR-Cas9 and autoimmune diseases: A comprehensive review. International Journal of Molecular Sciences, 23(3):1337. https://doi.org/10.3390/ijms23031337
Looney, W.J., Narita, M., and Mühlemann, K. (2019). Stenotrophomonas maltophilia: an emerging opportunist human pathogen. Lancet Infectious Diseases, The, 9(5):312-323. https://doi.org/10.1016/S1473-3099(09)70083-0
Lu, L., Yu, X., Cai, Y., Sun, M., and Yang, H. (2021). Application of CRISPR/Cas9 in Alzhiemer’s disease. Frontiers in Neuroscience, 15:8038894. https://doi.org/10.3389/fnins. 2021.803894
Maxwell, K.G., Augsornworawat, P., Velazco-Cruz, L., Kim, M.H., Asada, R., Hogrebe, N.J., Morikawa, S., Urano, F., and Millman, J.R. (2020). Gene-edited human stem cell-derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice. Science Translational Medicine, 12(540):9106. https://doi.org/10.1126/scitranslmed. aax9106
Mehmood, A., Ali, W., Din, Z.U., Song, S., Sohail, M., Shah, W., Guo, J., Guo, R.-Y., Ilahi, I., Shah, S., Al-Shaebi, F., Zeb, L., Asiamah, E.A., Al-Dhamin Z., Bilal, H., and Li, B. (2021). Clustered regularly interspaced short palindromic repeats as an advanced treatment for Parkinson’s disease. Brain and Behavior, 11(8):e2280. https://doi.org/10.1002/ brb3.2280
Morishige, S., Mizuno, S., Ozawa, H., Nakamura, T., Mazahery, A., Nomura, K., Seki, R., Mouri, F., Osaki, K., Yamamura, K., Okamura, T., and Nagafuji, K. (2020). CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs. International Journal of Hematology, 111:225-233. https://doi.org/10.1007/s12185-019-02765-0
Paschon, V., Correia, F.F., Morena, B.C., da Silva, V.A., dos Santos, G.B., da Silva, M.C.C., Cristante, A.F., Willerth, S.M., Perrin, F.E., and Kihara, A.H. (2020). CRISPR, prime Editing, optogenetics, and DREADDs: New therapeutic approaches provided by emerging technologies in the treatment of spinal cord injury. Molecular Neurobiology, 57:2085-2100. https://doi.org/10.1007/s12035-019-01861-w
Shin, J., Miller, M., and Wang, Y.C. (2022). Recent advances in CRISPR-based systems for the detection of foodborne pathogens. Comprehensive Reviews in Food Science and Food Safety, 21(3):3010-3029. https://doi.org/10.1111/ 1541-4337.12956
Simonelli, F., Maguire, A.M., Testa, F., Pierce, E.A., Mingozzi, F., Bennicelli, J.L., Rossi, S., Marshall, K., Banfi, S., Surace, E.M., Sun, J., Redmond, T.M., Zhu, X., Shindler, K.S., Ying, G.-S., Carmela Ziviello, Carmela Acerra, J Fraser Wright, Jennifer Wellman McDonnell, Katherine A High, Jean Bennett, Alberto Auricchio. (2010). Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Molecular Therapy, 18(3):643-650. https://doi.org/10.1038/mt.2009.277
Sollelis, L., Ghorbal, M., MacPherson, C.Ross R., Martins, M., Kuk, N., Crobu, L., Bastien, P., Scherf, A., Lopez-Rubio, J.-J., and Sterkers, Y. (2015). First efficient CRISPR-Cas9-mediated genome editing in Leishmania parasites. Cellular Microbiology, 17(10):1405-1412. https://doi.org/10.1111/ cmi.12456
Sredni, S.T., Suzuki, M., Yang, J.-P., Topczewski, J., Bailey, A.W., Gokirmak, T., Gross, J.N., de Andrade, A., Kondo, A., Piper, D.R., and Tomita, T. (2017). A functional screening of the kinome identifies the Polo-like kinase 4 as a potential therapeutic target for malignant rhabdoid tumors, and possibly, other embryonal tumors of the brain. Pediatric Blood and Cancer, 64:e26551. https://doi.org/10.1002/ pbc.26551
Uddin, F., Rudin, C.M., and Sen, T. (2020). CRISPR gene therapy: applications, limitations, and implications for the future. Frontiers in Oncology, 10:1387. https://doi.org/10.3389/ fonc.2020.01387
Vachey, G. and Déglon, N. (2018). CRISPR/Cas9-mediated genome editing for Huntington’s disease. In: Methods in Molecular Biology, Clifton, N.J. (ed.). 1780:463-481. https://doi.org/ 10.1007/978-1-4939-7825-0_21
Wang, S., Min, Z., Ji, Q., Geng, L., Su, Y., Liu, Z., Hu, H., Wang, L., Zhang, W., Suzuiki, K., Huang, Y., Zhang, P., Tang, T.-S., Qu, J., Yu, Y., Liu, G.-H., and Qiao, J. (2020). Rescue of premature aging defects in Cockayne syndrome stem cells by CRISPR/Cas9-mediated gene correction. Protein & Cell, 11(1):1-22. https://doi.org/10.1007/s13238-019-0623-2
Xiao, Q., Guo, D., and Chen, S. (2019). Application of CRISPR/Cas9-based gene editing in HIV-1/AIDS therapy. Frontiers in Cellular and Infection Microbiology, 9:69. https://doi.org/10.3389/fcimb.2019.00069
Yadav, N., Narang, J., Chhillar, A.K., and Rana, J.S. (2021). CRISPR: A new paradigm of theranostics. Nanomedicine: Nanotechnology, Biology, and Medicine, 33:102350. https://doi.org/10.1016/j.nano.2020.102350








