CRISPR-Cas Technology As a Revolutionary Genome Editing Tool: Mechanisms and Biomedical Applications

Overview

Journal Iran Biomed J

Specialties Biology
General Medicine

Date 2023 Oct 24

PMID 37873636

Authors

S Ebrahimi

Mohammad Ali Khosravi

Abbasali Raz

Morteza Karimipoor

Parviz Parvizi

Affiliations

Soon will be listed here.

Abstract

Programmable nucleases are powerful genomic tools for precise genome editing. These tools precisely recognize, remove, or change DNA at a defined site, thereby, stimulating cellular DNA repair pathways that can cause mutations or accurate replacement or deletion/insertion of a sequence. CRISPR-Cas9 system is the most potent and useful genome editing technique adapted from the defense immune system of certain bacteria and archaea against viruses and phages. In the past decade, this technology made notable progress, and at present, it has largely been used in genome manipulation to make precise gene editing in plants, animals, and human cells. In this review, we aim to explain the basic principle, mechanisms of action, and applications of this system in different areas of medicine, with emphasizing on the detection and treatment of parasitic diseases.

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References

Huang X, Wang Y, Yan W, Smith C, Ye Z, Wang J . Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation. Stem Cells. 2015; 33(5):1470-9. PMC: 4628786. DOI: 10.1002/stem.1969. View

Hille F, Charpentier E . CRISPR-Cas: biology, mechanisms and relevance. Philos Trans R Soc Lond B Biol Sci. 2016; 371(1707). PMC: 5052741. DOI: 10.1098/rstb.2015.0496. View

Sanchez-Rivera F, Papagiannakopoulos T, Romero R, Tammela T, Bauer M, Bhutkar A . Rapid modelling of cooperating genetic events in cancer through somatic genome editing. Nature. 2014; 516(7531):428-31. PMC: 4292871. DOI: 10.1038/nature13906. View

Boch J, Scholze H, Schornack S, Landgraf A, Hahn S, Kay S . Breaking the code of DNA binding specificity of TAL-type III effectors. Science. 2009; 326(5959):1509-12. DOI: 10.1126/science.1178811. View

Feng W, Zong W, Wang F, Ju S . Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2): a review. Mol Cancer. 2020; 19(1):100. PMC: 7264920. DOI: 10.1186/s12943-020-01218-1. View

Vierstra J, Reik A, Chang K, Stehling-Sun S, Zhou Y, Hinkley S . Functional footprinting of regulatory DNA. Nat Methods. 2015; 12(10):927-30. PMC: 5381659. DOI: 10.1038/nmeth.3554. View

El Refaey M, Xu L, Gao Y, Canan B, Adesanya T, Warner S . In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice. Circ Res. 2017; 121(8):923-929. PMC: 5623072. DOI: 10.1161/CIRCRESAHA.117.310996. View

Zhang W, Karmakar S, Gannavaram S, Dey R, Lypaczewski P, Ismail N . A second generation leishmanization vaccine with a markerless attenuated Leishmania major strain using CRISPR gene editing. Nat Commun. 2020; 11(1):3461. PMC: 7351751. DOI: 10.1038/s41467-020-17154-z. View

Karvelis T, Gasiunas G, Miksys A, Barrangou R, Horvath P, Siksnys V . crRNA and tracrRNA guide Cas9-mediated DNA interference in Streptococcus thermophilus. RNA Biol. 2013; 10(5):841-51. PMC: 3737341. DOI: 10.4161/rna.24203. View

10.

Peng D, Kurup S, Yao P, Minning T, Tarleton R . CRISPR-Cas9-mediated single-gene and gene family disruption in Trypanosoma cruzi. mBio. 2015; 6(1):e02097-14. PMC: 4281920. DOI: 10.1128/mBio.02097-14. View

11.

Strecker J, Jones S, Koopal B, Schmid-Burgk J, Zetsche B, Gao L . Engineering of CRISPR-Cas12b for human genome editing. Nat Commun. 2019; 10(1):212. PMC: 6342934. DOI: 10.1038/s41467-018-08224-4. View

12.

Perez-Pinera P, Kocak D, Vockley C, Adler A, Kabadi A, Polstein L . RNA-guided gene activation by CRISPR-Cas9-based transcription factors. Nat Methods. 2013; 10(10):973-6. PMC: 3911785. DOI: 10.1038/nmeth.2600. View

13.

Cavazzana M, Antoniani C, Miccio A . Gene Therapy for β-Hemoglobinopathies. Mol Ther. 2017; 25(5):1142-1154. PMC: 5417842. DOI: 10.1016/j.ymthe.2017.03.024. View

14.

Carroll D . Genome engineering with zinc-finger nucleases. Genetics. 2011; 188(4):773-82. PMC: 3176093. DOI: 10.1534/genetics.111.131433. View

15.

Li H, Bello A, Smith G, Kielich D, Strong J, Pickering B . Degenerate sequence-based CRISPR diagnostic for Crimean-Congo hemorrhagic fever virus. PLoS Negl Trop Dis. 2022; 16(3):e0010285. PMC: 8939784. DOI: 10.1371/journal.pntd.0010285. View

16.

Bjursell M, Porritt M, Ericson E, Taheri-Ghahfarokhi A, Clausen M, Magnusson L . Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates α1-antitrypsin Deficiency Phenotype. EBioMedicine. 2018; 29:104-111. PMC: 5925576. DOI: 10.1016/j.ebiom.2018.02.015. View

17.

van Embden J, Cave M, Crawford J, Dale J, Eisenach K, Gicquel B . Strain identification of Mycobacterium tuberculosis by DNA fingerprinting: recommendations for a standardized methodology. J Clin Microbiol. 1993; 31(2):406-9. PMC: 262774. DOI: 10.1128/jcm.31.2.406-409.1993. View

18.

Hou Z, Zhang Y, Propson N, Howden S, Chu L, Sontheimer E . Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis. Proc Natl Acad Sci U S A. 2013; 110(39):15644-9. PMC: 3785731. DOI: 10.1073/pnas.1313587110. View

19.

Xu J, Peng C, Sankaran V, Shao Z, Esrick E, Chong B . Correction of sickle cell disease in adult mice by interference with fetal hemoglobin silencing. Science. 2011; 334(6058):993-6. PMC: 3746545. DOI: 10.1126/science.1211053. View

20.

Rey M, Bonk M, Hadjiliadis D . Cystic Fibrosis: Emerging Understanding and Therapies. Annu Rev Med. 2018; 70:197-210. DOI: 10.1146/annurev-med-112717-094536. View