Gene Editing on Center Stage - PubMed

Affiliations

• PMID: 29908711
• DOI: 10.1016/j.tig.2018.05.004

Review

Gene Editing on Center Stage

Rasmus O Bak et al. Trends Genet. 2018 Aug.

Abstract

Smithies et al. (1985) and Jasin and colleagues (1994) provided proof of concept that homologous recombination (HR) could be applied to the treatment of human disease and that its efficiency could be improved by the induction of double-strand breaks (DSBs). A key advance was the discovery of engineered nucleases, such as zinc-finger nucleases (ZFNs) and transcription activator-like (TAL) effector nucleases (TALENs), that can generate site-specific DSBs. The democratization and widespread use of genome editing was enabled by the discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease system. While genome editing using ZFNs and TALENs has already reached clinical trials, the pace at which genome editing enters human trials is bound to accelerate in the next several years with multiple promising preclinical studies heralding cures for monogenic diseases that are currently difficult to manage or even incurable. Here we review recent advances and current limitations and discuss the path forward using genome editing to understand, treat, and cure genetic diseases.

Keywords: CRISPR–Cas9; gene therapy; genome editing; homologous recombination.

Similar articles

• Therapeutic Genome Editing and its Potential Enhancement through CRISPR Guide RNA and Cas9 Modifications.

  Batzir NA, Tovin A, Hendel A. Batzir NA, et al. Pediatr Endocrinol Rev. 2017 Jun;14(4):353-363. doi: 10.17458/per.vol14.2017.BTH.Therapeu. Pediatr Endocrinol Rev. 2017. PMID: 28613045 Review.

• INDEL detection, the 'Achilles heel' of precise genome editing: a survey of methods for accurate profiling of gene editing induced indels.

  Bennett EP, Petersen BL, Johansen IE, Niu Y, Yang Z, Chamberlain CA, Met Ö, Wandall HH, Frödin M. Bennett EP, et al. Nucleic Acids Res. 2020 Dec 2;48(21):11958-11981. doi: 10.1093/nar/gkaa975. Nucleic Acids Res. 2020. PMID: 33170255 Free PMC article. Review.

• A beginner's guide to gene editing.

  Harrison PT, Hart S. Harrison PT, et al. Exp Physiol. 2018 Apr 1;103(4):439-448. doi: 10.1113/EP086047. Epub 2018 Jan 25. Exp Physiol. 2018. PMID: 29282799 Review.

• CRISPR/Cas9 gene-editing strategies in cardiovascular cells.

  Vermersch E, Jouve C, Hulot JS. Vermersch E, et al. Cardiovasc Res. 2020 Apr 1;116(5):894-907. doi: 10.1093/cvr/cvz250. Cardiovasc Res. 2020. PMID: 31584620 Review.

• Genetic Modification in Human Pluripotent Stem Cells by Homologous Recombination and CRISPR/Cas9 System.

  Xue H, Wu J, Li S, Rao MS, Liu Y. Xue H, et al. Methods Mol Biol. 2016;1307:173-90. doi: 10.1007/7651_2014_73. Methods Mol Biol. 2016. PMID: 24615461

Cited by

• Genome-Editing Products Line up for the Market: Will Europe Harvest the Benefits from Science and Innovation?

  Polidoros A, Nianiou-Obeidat I, Tsakirpaloglou N, Petrou N, Deligiannidou E, Makri NM. Polidoros A, et al. Genes (Basel). 2024 Aug 1;15(8):1014. doi: 10.3390/genes15081014. Genes (Basel). 2024. PMID: 39202374 Free PMC article. Review.

• Multiplex CRISPR/Cas9-mediated metabolic engineering increases soya bean isoflavone content and resistance to soya bean mosaic virus.

  Zhang P, Du H, Wang J, Pu Y, Yang C, Yan R, Yang H, Cheng H, Yu D. Zhang P, et al. Plant Biotechnol J. 2020 Jun;18(6):1384-1395. doi: 10.1111/pbi.13302. Epub 2019 Dec 9. Plant Biotechnol J. 2020. PMID: 31769589 Free PMC article.

• Deep Characterization and Comparison of Different Retrovirus-like Particles Preloaded with CRISPR/Cas9 RNPs.

  Wichmann M, Maire CL, Nuppenau N, Habiballa M, Uhde A, Kolbe K, Schröder T, Lamszus K, Fehse B, Głów D. Wichmann M, et al. Int J Mol Sci. 2023 Jul 13;24(14):11399. doi: 10.3390/ijms241411399. Int J Mol Sci. 2023. PMID: 37511168 Free PMC article.

• Understanding Non-Mendelian Genetic Risk.

  Coetzee GA. Coetzee GA. Curr Genomics. 2019 Aug;20(5):322-324. doi: 10.2174/1389202920666191018085511. Curr Genomics. 2019. PMID: 32476988 Free PMC article. Review.

• CRISPR/Cas Systems in Genome Editing: Methodologies and Tools for sgRNA Design, Off-Target Evaluation, and Strategies to Mitigate Off-Target Effects.

  Manghwar H, Li B, Ding X, Hussain A, Lindsey K, Zhang X, Jin S. Manghwar H, et al. Adv Sci (Weinh). 2020 Feb 6;7(6):1902312. doi: 10.1002/advs.201902312. eCollection 2020 Mar. Adv Sci (Weinh). 2020. PMID: 32195078 Free PMC article. Review.

Publication types

MeSH terms

Substances

LinkOut - more resources

• Full Text Sources

  • ClinicalKey
  • Elsevier Science

• Other Literature Sources

  • scite Smart Citations

• Miscellaneous

  • NCI CPTAC Assay Portal