Applications of CRISPR technologies to the development of gene and cell therapy

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참고문헌 (88)

1. Kim H, "A guide to genome engineering with programmable nucleases", Nat Rev Genet, 15, pp.321~334 (2014)
2. Lee SH, "CRISPR diagnosis and therapeutics with single base pair precision", Trends Mol Med, 26, pp.337~350 (2020)
3. Wright AV, "Biology and applications of CRISPR systems : harnessing nature’s toolbox for genome engineering", Cell, 164, pp.29~44 (2016)
4. Jinek M, "A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity", Science, 337, pp.816~821 (2012)
5. Hsu PD, "Development and applications of CRISPR-Cas9 for genome engineering", Cell, 157, pp.1262~1278 (2014)
6. Lee SH, "CRISPR and targetspecific DNA endonucleases for efficient DNA knock-in in eukaryotic genomes", Mol Cells, 41, pp.943~952 (2018)
7. Zhang XH, "Off-target effects in CRISPR", Cas9-mediated genome engineering, 2015, pp.4, e264~
8. Liu Y, "Global chromosome rearrangement induced by CRISPR-Cas9 reshapes the genome and transcriptome of human cells", Nucleic Acids Res, 50, pp.3456~3474 (2022)
9. Alvarez MM, "TP53-dependent toxicity of CRISPR", Cas9 cuts is differential across genomic loci and can confound genetic screening, 2022, pp.13, 4520~
10. Tsai SQ, "Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases", Nat Rev Genet, 17, pp.300~312 (2016)
11. Kang SH, "Prediction-based highly sensitive CRISPR off-target validation using targetspecific DNA enrichment", Nat Commun, 11, pp.3596~ (2020)
12. Yen ST, "Somatic mosaicism and allele complexity induced by CRISPR", Cas9 RNA injections in mouse zygotes, 2014, pp.393, 3~9
13. 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
14. Lamas-Toranzo I, "Strategies to reduce genetic mosaicism following CRISPR-mediated genome edition in bovine embryos", Sci Rep, 9, pp.14900~ (2019)
15. Mehravar M, "Mosaicism in CRISPR", Cas9-mediated genome editing, 2019, pp.445, 156~162
16. Yeh CD, "Advances in genome editing through control of DNA repair pathways", Nat Cell Biol, 21, pp.1468~1478 (2019)
17. Landrum MJ, "ClinVar : public archive of interpretations of clinically relevant variants", Nucleic Acids Res, 44, pp.D862~868 (2016)
18. Komor AC, "Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage", Nature, 533, pp.420~424 (2016)
19. Gaudelli NM, "Programmable base editing of A*T to G*C in genomic DNA without DNA cleavage", Nature, 551, pp.464~471 (2017)
20. Anzalone AV, "Search-and-replace genome editing without double-strand breaks or donor DNA", Nature, 576, pp.149~157 (2019)
21. Kim S, "Targeted genome engineering via zinc finger nucleases", Plant Biotechnol Rep, 5, pp.9~17 (2011)
22. Ul Ain Q, "Current and future delivery systems for engineered nucleases : ZFN, TALEN and RGEN", J Control Release, 205, pp.120~127 (2015)
23. Petersen B, "Basics of genome editing technology and its application in livestock species", Reprod Domest Anim 52 Suppl, 3, pp.4~13 (2017)
24. Nemudryi AA, "TALEN and CRISPR", Cas genome editing systems : tools of discovery, 2014, pp.6, 19~40
25. Mali P, "RNA-guided human genome engineering via Cas9", Science, 339, pp.823~826 (2013)
26. Cong L, "Multiplex genome engineering using CRISPR", Cas systems, 2013, pp.339, 819~823
27. Huang CJ, "A naturally DNase-free CRISPR-Cas12c enzyme silences gene expression", Mol Cell, 82, pp.2148~2160 (2022)
28. Kato K, "Structure and engineering of the type III-E CRISPR-Cas7-11 effector complex", Cell, 185, pp.2324~2337 (2022)
29. Jiang F, "CRISPR-Cas9 Structures and Mechanisms", Annu Rev Biophys, 46, pp.505~529 (2017)
30. Nishimasu H, "Crystal structure of Cas9 in complex with guide RNA and target DNA", Cell, 156, pp.935~949 (2014)
31. Jiang F, "The structural biology of CRISPR-Cas systems", Curr Opin Struct Biol, 30, pp.100~111 (2015)
32. Jiang F, "STRUCTURAL BIOLOGY. A Cas9-guide RNA complex preorganized for target DNA recognition", Science, 348, pp.1477~1481 (2015)
33. Jiang F, "Structures of a CRISPR-Cas9 R-loop complex primed for DNA cleavage", Science, 351, pp.867~871 (2016)
34. Hsu PD, "DNA targeting specificity of RNA-guided Cas9 nucleases", Nat Biotechnol, 31, pp.827~832 (2013)
35. Fonfara I, "Phylogeny of Cas9 determines functional exchangeability of dual-RNA and Cas9 among orthologous type II CRISPR-Cas systems", Nucleic Acids Res, 42, pp.2577~2590 (2014)
36. Anders C, "Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease", Nature, 513, pp.569~573 (2014)
37. Hu JH, "Evolved Cas9variants with broad PAM compatibility and high DNA specificity", Nature, 556, pp.57~63 (2018)
38. Kleinstiver BP, "Engineered CRISPR-Cas9 nucleases with altered PAM specificities", Nature, 523, pp.481~485 (2015)
39. Li X, "Homologous recombination in DNA repair and DNA damage tolerance", Cell Res, 18, pp.99~113 (2008)
40. Lieber MR, "The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway", Annu Rev Biochem, 79, pp.181~211 (2010)
41. Sfeir A, "Microhomology-mediated end joining : a back-up survival mechanism or dedicated pathway?", Trends Biochem Sci, 40, pp.701~714 (2015)
42. Chapman JR, "Playing the end game : DNA double-strand break repair pathway choice", Mol Cell, 47, pp.497~510 (2012)
43. Shrivastav M, "Regulation of DNA double-strand break repair pathway choice", Cell Res, 18, pp.134~147 (2008)
44. Deriano L, "Modernizing the nonhomologous end-joining repertoire : alternative and classical NHEJ share the stage", Annu Rev Genet, 47, pp.433~455 (2013)
45. Heyer WD, "Regulation of homologous recombination in eukaryotes", Annu Rev Genet, 44, pp.113~139 (2010)
46. Krejci L, "Homologous recombination and its regulation", Nucleic Acids Res, 40, pp.5795~5818 (2012)
47. Sakuma T, "MMEJ-assisted gene knock-in using TALENs and CRISPR-Cas9 with the PITCh systems", Nat Protoc, 11, pp.118~133 (2016)
48. Bae S, "Microhomology-based choice of Cas9 nuclease target sites", Nat Methods, 11, pp.705~706 (2014)
49. Cho SW, "Targeted genome engineering in human cells with the Cas9 RNAguided endonuclease", Nat Biotechnol, 31, pp.230~232 (2013)
50. Scharenberg SG, "Engineering monocyte", macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing, 2020, pp.11, 3327~
51. Maxwell KG, "Gene-edited human stem cell-derived beta cells from a patient with monogenic diabetes reverse preexisting diabetes in mice", Sci Transl Med, 12, pp.9106~ (2020)
52. Smith C, "Efficient and allele-specific genome editing of disease loci in human iPSCs", Mol Ther, 23, pp.570~577 (2015)
53. Shin JW, "Permanent inactivation of Huntington’s disease mutation by personalized allele-specific CRISPR", Cas9, 2016, pp.25, 4566~4576
54. Monteys AM, "CRISPR", Cas9 editing of the mutant huntingtin allele in vitro and in vivo, 2017, pp.25, 12~23
55. Cai W, "Orthogonal chemical activation of enzyme-inducible CRISPR", Cas9 for cell-selective genome editing, 2022, pp.144, 22272~22280
56. Frangoul H, "CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia", N Engl J Med, 384, pp.252~260 (2020)
57. Sheridan C, "The world’s first CRISPR therapy is approved: who will receive it?", Nat Biotechnol (2023)
58. Gillmore JD, "CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis", N Engl J Med, 385, pp.493~502 (2021)
59. Christie KA, "Towards personalised allele-specific CRISPR gene editing to treat autosomal dominant disorders", Sci Rep, 7, pp.16174~ (2017)
60. Li P, "Allele-Specific CRISPR-Cas9 genome editing of the single-base P23H mutation for rhodopsin-associated dominant Retinitis Pigmentosa", CRISPR J, 1, pp.55~64 (2018)
61. Rees HA, "Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks", Nat Commun, 10, pp.2212~ (2019)
62. Ling X, "Improving the efficiency of precise genome editing with site-specific Cas9-oligonucleotide conjugates", Sci Adv, 6, pp.51~ (2020)
63. Yu Y, "An efficient gene knock-in strategy using 5’-modified double-stranded DNA donors with short homology arms", Nat Chem Biol, 16, pp.387~390 (2020)
64. Nguyen DN, "Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency", Nat Biotechnol, 38, pp.44~49 (2020)
65. Egli D, "Inter-homologue repair in fertilized human eggs?", Nature, 560, pp.E5~E7 (2018)
66. Nishida K, "Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems", Science, 353, pp.8729~ (2016)
67. 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.4774~ (2017)
68. Zafra MP, "Optimized base editors enable efficient editing in cells, organoids and mice", Nat Biotechnol, 36, pp.888~893 (2018)
69. Koblan LW, "Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction", Nat Biotechnol, 36, pp.843~846 (2018)
70. Kurt IC, "CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells", Nat Biotechnol, 39, pp.41~46 (2020)
71. Lin Q, "Prime genome editing in rice and wheat", Nat Biotechnol, 38, pp.582~585 (2020)
72. Sürün D, "Efficient generation and correction of mutations in human iPS cells utilizing mRNAs of CRISPR base editors and prime editors", Genes, 11, pp.511~ (2020)
73. Liu Y, "Efficient generation of mouse models with the prime editing system", Cell Discov, 6, pp.27~ (2020)
74. Chen PJ, "Enhanced prime editing systems by manipulating cellular determinants of editing outcomes", Cell, 184, pp.5635~5652 (2021)
75. Park SJ, "Targeted mutagenesis in mouse cells and embryos using an enhanced prime editor", Genome Biol, 22, pp.170~ (2021)
76. Yarnall MTN, "Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases", Nat Biotechnol, 41, pp.500~512 (2023)
77. Yeh WH, "In vivo base editing of post-mitotic sensory cells", Nat Commun, 9, pp.2184~ (2018)
78. Arbab M, "Base editing rescue of spinal muscular atrophy in cells and in mice", Science, 380, pp.6518~ (2023)
79. Ryu SM, "Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy", Nat Biotechnol, 36, pp.536~539 (2018)
80. Gehrke JM, "An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities", Nat Biotechnol, 36, pp.977~982 (2018)
81. Rovai A, "In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice", Nat Commun, 13, pp.5215~ (2022)
82. McAuley GE, "Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing", Cell, 186, pp.1398~1416 (2023)
83. Reichart D, "Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice", Nat Med, 29, pp.412~421 (2023)
84. Hong SA, "Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa", Mol Ther, 30, pp.2664~2679 (2022)
85. Jang H, "Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases", Nat Biomed Eng, 6, pp.181~194 (2022)
86. Qin H, "Vision rescue via unconstrained in vivo prime editing in degenerating neural retinas", J Exp Med, 220, pp.e20220776~ (2023)
87. Walton RT, "Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants", Science, 368, pp.290~296 (2020)
88. Jang G, "CRISPR prime editing for unconstrained correction of oncogenic KRAS variants", Commun Biol, 6, pp.681~ (2023)

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