Development of CRISPR technology for precise single-base genome editing: a brief review
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The clustered regularly interspaced short palindromic repeats (CRISPR) system is a family of DNA sequences originally discovered as a type of acquired immunity in prokaryotes such as bacteria and archaea. In many CRISPR systems, the functional ribonucleoproteins (RNPs) are composed of CRISPR protein and guide RNAs. They selectively bind and cleave specific target DNAs or RNAs, based on sequences complementary to the guide RNA. The specific targeted cleavage of the nucleic acids by CRISPR has been broadly utilized in genome editing methods. In the process of genome editing of eukaryotic cells, CRISPRmediated DNA double-strand breaks (DSB) at specific genomic loci activate the endogenous DNA repair systems and induce mutations at the target sites with high efficiencies. Two of the major endogenous DNA repair machineries are non-homologous end joining (NHEJ) and homology-directed repair (HDR). In case of DSB, the two repair pathways operate in competition, resulting in several possible outcomes including deletions, insertions, and substitutions. Due to the inherent stochasticity of DSB-based genome editing methods, it was difficult to achieve defined single-base changes without unanticipated random mutation patterns. In order to overcome the heterogeneity in DSB-mediated genome editing, novel methods have been developed to incorporate precise single-base level changes without inducing DSB. The approaches utilized catalytically compromised CRISPR in conjunction with base-modifying enzymes and DNA polymerases, to accomplish highly efficient and precise genome editing of single and multiple bases. In this review, we introduce some of the advances in single-base level CRISPR genome editing methods and their applications.
Abstract
The clustered regularly interspaced short palindromic repeats (CRISPR) system is a family of DNA sequences originally discovered as a type of acquired immunity in prokaryotes such as bacteria and archaea. In many CRISPR systems, the functional ribonucleoproteins (RNPs) are composed of CRISPR protein and guide RNAs. They selectively bind and cleave specific target DNAs or RNAs, based on sequences complementary to the guide RNA. The specific targeted cleavage of the nucleic acids by CRISPR has been broadly utilized in genome editing methods. In the process of genome editing of eukaryotic cells, CRISPRmediated DNA double-strand breaks (DSB) at specific genomic loci activate the endogenous DNA repair systems and induce mutations at the target sites with high efficiencies. Two of the major endogenous DNA repair machineries are non-homologous end joining (NHEJ) and homology-directed repair (HDR). In case of DSB, the two repair pathways operate in competition, resulting in several possible outcomes including deletions, insertions, and substitutions. Due to the inherent stochasticity of DSB-based genome editing methods, it was difficult to achieve defined single-base changes without unanticipated random mutation patterns. In order to overcome the heterogeneity in DSB-mediated genome editing, novel methods have been developed to incorporate precise single-base level changes without inducing DSB. The approaches utilized catalytically compromised CRISPR in conjunction with base-modifying enzymes and DNA polymerases, to accomplish highly efficient and precise genome editing of single and multiple bases. In this review, we introduce some of the advances in single-base level CRISPR genome editing methods and their applications.
참고문헌 (49)
- Kim H, "A guide to genome engineering with programmable nucleases", Nat Rev Genet, 15, pp.321~334 (2014)
- Lee SH, "CRISPR diagnosis and therapeutics with single base pair precision", Trends Mol Med, 26, pp.337~350 (2020)
- Wright AV, "Biology and applications of CRISPR systems : harnessing nature’s toolbox for genome engineering", Cell, 164, pp.29~44 (2016)
- Hsu PD, "Development and applications of CRISPR-Cas9 for genome engineering", Cell, 157, pp.1262~1278 (2014)
- Jinek M, "A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity", Science, 337, pp.816~821 (2012)
- 이승환, "CRISPR and Target-Specific DNA Endonucleases for Efficient DNA Knock-in in Eukaryotic Genomes", Molecules and Cells, 41(11), pp.943~952 (2018), 한국분자세포생물학회[DOI]
- Tsai SQ, "Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases", Nat Rev Genet, 17, pp.300~312 (2016)
- Yen ST, "Somatic mosaicism and allele complexity induced by CRISPR", Cas9 RNA injections in mouse zygotes, 2014, pp.393, 3~9
- Midic U, "Quantitative assessment of timing, efficiency, specificity and genetic mosaicism of CRISPR", Cas9-mediated gene editing of hemoglobin beta gene in rhesus monkey embryos, 2017, pp.26, 2678~2689
- Lamas-Toranzo I, "Strategies to reduce genetic mosaicism following CRISPR-mediated genome edition in bovine embryos", Sci Rep, 9, pp.14900~ (2019)
- Mehravar M, "Mosaicism in CRISPR", Cas9-mediated genome editing, 2019, pp.445, 156~162
- Yeh CD, "Advances in genome editing through control of DNA repair pathways", Nat Cell Biol, 21, pp.1468~1478 (2019)
- Landrum MJ, "ClinVar : public archive of interpretations of clinically relevant variants", Nucleic Acids Res, 44, pp.D862~D868 (2016)
- Rees HA, "Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks", Nat Commun, 10, pp.2212~ (2019)
- Ling X, "Improving the efficiency of precise genome editing with site-specific Cas9-oligonucleotide conjugates", Sci Adv, 6, pp.eaaz0051~ (2020)
- Yu Y, "An efficient gene knockn strategy using 5’odified double-stranded DNA donors with short homology arms", Nat Chem Biol, 16, pp.387~390 (2020)
- Nguyen DN, "Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency", Nat Biotechnol, 38, pp.44~49 (2020)
- Egli D, "Inter-homologue repair in fertilized human eggs?", Nature, 560, pp.E5~E7 (2018)
- Komor AC, "Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage", Nature, 533, pp.420~424 (2016)
- Gaudelli NM, "Programmable base editing of A*T to G*C in genomic DNA without DNA cleavage", Nature, 551, pp.464~471 (2017)
- Anzalone AV, "Searchnd-replace genome editing without double-strand breaks or donor DNA", Nature, 576, pp.149~157 (2019)
- Komor AC, "Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C : G-to-T : A base editors with higher efficiency and product purity", Sci Adv, 3, pp.eaao4774~ (2017)
- Zafra MP, "Optimized base editors enable efficient editing in cells, organoids and mice", Nat Biotechnol, 36, pp.888~893 (2018)
- Koblan LW, "Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction", Nat Biotechnol, 36, pp.843~846 (2018)
- Kurt IC, "CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells", Nat Biotechnol, 39, pp.41~46 (2021)
- Kim J, "Structural and kinetic characterization of Escherichia coli TadA, the wobble-specific tRNA deaminase", Biochemistry, 45, pp.6407~6416 (2006)
- Richter MF, "Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity", Nat Biotechnol, 38, pp.883~891 (2020)
- Ryu SM, "Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy", Nat Biotechnol, 36, pp.536~539 (2018)
- Lee HK, "Targeting fidelity of adenine and cytosine base editors in mouse embryos", Nat Commun, 9, pp.4804~ (2018)
- Rees HA, "Analysis and minimization of cellular RNA editing by DNA adenine base editors", Sci Adv, 5, pp.eaax5717~ (2019)
- Kim YB, "Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions", Nat Biotechnol, 35, pp.371~376 (2017)
- Hu JH, "Evolved Cas9variants with broad PAM compatibility and high DNA specificity", Nature, 556, pp.57~63 (2018)
- Huang TP, "Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors", Nat Biotechnol, 37, pp.626~631 (2019)
- Walton RT, "Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants", Science, 368, pp.290~296 (2020)
- Li X, "Base editing with a Cpf1-cytidine deaminase fusion", Nat Biotechnol, 36, pp.324~327 (2018)
- Kleinstiver BP, "Highfidelity CRISPR-Cas9 nucleases with no detectable genomewide off-target effects", Nature, 529, pp.490~495 (2016)
- Rees HA, "Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery", Nat Commun, 8, pp.15790~ (2017)
- Kim S, "Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins", Genome Res, 24, pp.1012~1019 (2014)
- Gehrke JM, "An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities", Nat Biotechnol, 36, pp.977~982 (2018)
- Yeh WH, "In vivo base editing of post-mitotic sensory cells", Nat Commun, 9, pp.2184~ (2018)
- Lee HK, "Simultaneous targeting of linked loci in mouse embryos using base editing", Sci Rep, 9, pp.1662~ (2019)
- Zong Y, "Precise base editing in rice, wheat and maize with a Cas9-cytidine deaminase fusion", Nat Biotechnol, 35, pp.438~440 (2017)
- Hua K, "Precise A. T to G. C base editing in the rice genome", Mol Plant, 11, pp.627~630 (2018)
- Ma Y, "Targeted AID-mediated mutagenesis(TAM)enables efficient genomic diversification in mammalian cells", Nat Methods, 13, pp.1029~1035 (2016)
- Hess GT, "Directed evolution using dCas9-targeted somatic hypermutation in mammalian cells", Nat Methods, 13, pp.1036~1042 (2016)
- Lin Q, "Prime genome editing in rice and wheat", Nat Biotechnol, 38, pp.582~585 (2020)
- Surun D, "Efficient generation and correction of mutations in human iPS cells utilizing mRNAs of CRISPR base editors and prime editors", Genes(Basel), 11, pp.511~ (2020)
- Liu Y, "Efficient generation of mouse models with the prime editing system", Cell Discov, 6, pp.27~ (2020)
- Clement K, "CRISPResso2provides accurate and rapid genome editing sequence analysis", Nat Biotechnol, 37, pp.224~226 (2019)
