CRISPR-Cas9 as a tool for treating cystic fibrosis through gene editing

Jiabao Xu

doi:10.54254/2753-8818/6/20230222

Research Article

Open access

Published on 3 August 2023

Download pdf

Xu,J. (2023). CRISPR-Cas9 as a tool for treating cystic fibrosis through gene editing. Theoretical and Natural Science,6,191-197.

Export citation

CRISPR-Cas9 as a tool for treating cystic fibrosis through gene editing

Jiabao Xu *^,1,

¹ Chongqing Depu Foreign Language School

* Author to whom correspondence should be addressed.

https://doi.org/10.54254/2753-8818/6/20230222

Abstract

The most common cause of mortality is Cystic Fibrosis, a fatal genetic disease. However, in recent years, there have been a growing number of papers concentrating on CRISPR-Cas9, a gene-editing tool that is being used to permanently cure this genetic disease, named by a biopharmaceutical company EditasMedicine, invested in by Bill Gates. However, before the breadth of search and study of this technology continuously expands, challenges and remaining issues should be addressed. This paper reviews the mechanisms of cystic fibrosis and discusses its technical challenges, such as efficiency, safety and delivery of gene editing, potential side effects, and ethical issues, this paper also talks about the future applications of CRISPR-Cas9 in other diseases, so as to provide an alternative treatment method for the diseases with gene editing better results can be obtained.

Keywords

cystic fibrosis, CRISPR-Cas9, gene edit, CFTR

View pdf

References

[1]. di Sant'Agnese, P. A. Fibrocystic disease of the pancreas, a generalized disease of exocrine glands. Journal of the American Medical Association, 160(10), 846-853 (1956).

[2]. Park, H., Oh, J., Shim, G., Cho, B., Chang, Y., Kim, S., & Baek, S. In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease. Nature Neuroscience, 22(4), 524+ (2019). https://link.gale.com/apps/doc/A580134231/HRCA?u=anon~401fac63&sid=googleScholar&xid=0d4e9e6e)

[3]. Davis, P. B. Cystic fibrosis since 1938. American journal of respiratory and critical care medicine, 173(5), 475-482 (2006).

[4]. Demirci S., Leonard A., Haro-Mora J.J., Uchida N., Tisdale J.F. CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges. Adv Exp Med Biol. 1144:37-52 (2019). doi: 10.1007/5584_2018_331. PMID: 30715679.

[5]. Graham, C., & Hart, S. CRISPR/Cas9 gene editing therapies for cystic fibrosis. Expert Opinion on Biological Therapy, 21(6), 767-780 (2021).

[6]. Belkin, R. A., Henig, N. R., Singer, L. G., Chaparro, C., Rubenstein, R. C., Xie, S. X., ... & Bunin, G. R. Risk factors for death of patients with cystic fibrosis awaiting lung transplantation. American journal of respiratory and critical care medicine, 173(6), 659-666 (2006).

[7]. Murray, J. F. The ketchup-bottle method. New England Journal of Medicine, 300(20), 1155-1157 (1979).

[8]. Raman, V., Clary, R., Siegrist, K. L., Zehnbauer, B., & Chatila, T. A. Increased prevalence of mutations in the cystic fibrosis transmembrane conductance regulator in children with chronic rhinosinusitis. Pediatrics, 109(1), e13-e13 (2002).

[9]. Saiman, L., Marshall, B. C., Mayer-Hamblett, N., Burns, J. L., Quittner, A. L., Cibene, D. A., ... & Macrolide Study Group. Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial. Jama, 290(13), 1749-1756 (2003).

[10]. Ramsey, B. W., Dorkin, H. L., Eisenberg, J. D., Gibson, R. L., Harwood, I. R., Kravitz, R. M., ... & Smith, A. L. Efficacy of aerosolized tobramycin in patients with cystic fibrosis. New England Journal of Medicine, 328(24), 1740-1746 (1993).

[11]. Bell, S. C. The Future of Cystic Fibrosis Care: A Global Perspective. The Lancet Respiratory Medicine, 8(1), 65–124 (2019). https://doi.org/https://doi.org/10.1016/S2213-2600(19)30337-6

[12]. Gentzsch, M., & Mall, M. A. Ion channel modulators in cystic fibrosis. Chest, 154(2), 383-393 (2018).

[13]. Castellani, C., Cuppens, H., & Macek Jr, M. Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice. journal of cystic fibrosis, Off. J. Eur. Cyst. Fibros. Soc, 7 (2008).

[14]. Cutting, G., Sosnay, P., Karchin, R., Zielenski, J., Penland, C., & Castellani, C. CFTR2: Defining the clinical and functional consequences of CFTR mutations. Pediatric pulmonology, 44(SUPPL. 32), 161-162 (2009).

[15]. Bear, C. E. A therapy for most with cystic fibrosis. Cell, 180(2), 211 (2020).

[16]. Szczepek, M., Brondani, V., Büchel, J., Serrano, L., Segal, D. J., & Cathomen, T. Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. Nature biotechnology, 25(7), 786–793 (2007). https://doi.org/10.1038/nbt1317

[17]. Wood, A. J., Lo, T. W., Zeitler, B., Pickle, C. S., Ralston, E. J., Lee, A. H., Amora, R., Miller, J. C., Leung, E., Meng, X., Zhang, L., Rebar, E. J., Gregory, P. D., Urnov, F. D., & Meyer, B. J. Targeted genome editing across species using ZFNs and TALENs. Science (New York, N.Y.), 333(6040), 307 (2011). https://doi.org/10.1126/science.1207773

[18]. Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity, Science337: 816–821 (2012).

[19]. Barrangou, R., Fremaux, C., Deveau, H., Richards, M., Boyaval, P., Moineau, S., ... & Horvath, P. CRISPR provides acquired resistance against viruses in prokaryotes. Science, 315(5819), 1709-1712 (2007).

[20]. Mojica, F. J., & Montoliu, L. On the origin of CRISPR-Cas technology: from prokaryotes to mammals. Trends in microbiology, 24(10), 811-820 (2016).

[21]. Khan, F. A., Pandupuspitasari, N. S., Chun-Jie, H., Ao, Z., Jamal, M., Zohaib, A., Khan, F. A., Hakim, M. R., & ShuJun, Z. CRISPR/Cas9 therapeutics: a cure for cancer and other genetic diseases. Oncotarget, 7(32), 52541–52552 (2016). https://doi.org/10.18632/oncotarget.9646

[22]. Schwank, G., Koo, B. K., Sasselli, V., Dekkers, J. F., Heo, I., Demircan, T., ... & Clevers, H. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. Cell stem cell, 13(6), 653-658 (2013).

[23]. Chen, M., Mao, A., Xu, M., Weng, Q., Mao, J., & Ji, J. CRISPR-Cas9 for cancer therapy: Opportunities and challenges. Cancer letters, 447, 48–55 (2019). https://doi.org/10.1016/j.canlet.2019.01.017

[24]. Xue, W., Chen, S., Yin, H., Tammela, T., Papagiannakopoulos, T., Joshi, N. S., Cai, W., Yang, G., Bronson, R., Crowley, D. G., Zhang, F., Anderson, D. G., Sharp, P. A., & Jacks, T. CRISPR-mediated direct mutation of cancer genes in the mouse liver. Nature, 514(7522), 380–384 (2014). https://doi.org/10.1038/nature13589

[25]. Tsai, S. Q., & Joung, J. K. Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases. Nature Reviews Genetics, 17(5), 300-312 (2016).

[26]. Fu, Y., Foden, J. A., Khayter, C., Maeder, M. L., Reyon, D., Joung, J. K., & Sander, J. D. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nature biotechnology, 31(9), 822-826 (2013).

[27]. Sykora, P. Germline gene therapy in the era of precise genome editing: How far should we go?”. W: The Ethics of Reproductive Genetics: Between Utility, Principles, and Virtues, red. Marta Soniewicka, 187–202 (2018).

[28]. Wirth, T., Parker, N., & Ylä-Herttuala, S. History of gene therapy. Gene, 525(2), 162-169 (2013).

[29]. Hong, A. CRISPR in personalized medicine: Industry perspectives in gene editing. In Seminars in perinatology, WB Saunders, Vol. 42, No. 8, pp. 501-507 (2018).

[30]. Uddin, F., Rudin, C. M., & Sen, T. CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future. Frontiers in oncology, 10, 1387 (2020). https://doi.org/10.3389/fonc.2020.01387

[31]. Zych, A. O., Bajor, M., & Zagozdzon, R. Application of genome editing techniques in immunology. Archivum Immunologiae et Therapiae Experimentalis, 66, 289-298 (2018).

[32]. Cyranoski, D. Chinese scientists to pioneer first human CRISPR trial. Nature, 535(7613), 476-477 (2016).

Cite this article

Xu,J. (2023). CRISPR-Cas9 as a tool for treating cystic fibrosis through gene editing. Theoretical and Natural Science,6,191-197.

Data availability

The datasets used and/or analyzed during the current study will be available from the authors upon reasonable request.

Disclaimer/Publisher's Note

The statements, opinions and data contained in all publications are solely those of the individual author(s) and contributor(s) and not of EWA Publishing and/or the editor(s). EWA Publishing and/or the editor(s) disclaim responsibility for any injury to people or property resulting from any ideas, methods, instructions or products referred to in the content.

About volume

Volume title: Proceedings of the International Conference on Modern Medicine and Global Health (ICMMGH 2023)

Conference website: https://www.icmmgh.org/

ISBN：978-1-915371-65-2(Print) / 978-1-915371-66-9(Online)

Conference date: 15 April 2023

Editor：Tooba Mahboob, Sheiladevi Sukumaran

Series: Theoretical and Natural Science

Volume number: Vol.6

ISSN：2753-8818(Print) / 2753-8826(Online)

© 2024 by the author(s). Licensee EWA Publishing, Oxford, UK. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license. Authors who publish this series agree to the following terms:
1. Authors retain copyright and grant the series right of first publication with the work simultaneously licensed under a Creative Commons Attribution License that allows others to share the work with an acknowledgment of the work's authorship and initial publication in this series.
2. Authors are able to enter into separate, additional contractual arrangements for the non-exclusive distribution of the series's published version of the work (e.g., post it to an institutional repository or publish it in a book), with an acknowledgment of its initial publication in this series.
3. Authors are permitted and encouraged to post their work online (e.g., in institutional repositories or on their website) prior to and during the submission process, as it can lead to productive exchanges, as well as earlier and greater citation of published work (See Open access policy for details).