Mitochondrial genome editing: strategies, challenges, and applications

키워드

참고문헌 (72)

1. Rackham O, "Organization and expression of the mammalian mitochondrial genome", Nat Rev Genet, 23, pp.606~623 (2022)
2. Gupta R, "Nuclear genetic control of mtDNA copy number and heteroplasmy in humans", Nature, 620, pp.839~848 (2023)
3. Gorman GS, "Prevalence of nuclear and mitochondrial DNA mutations related to adult mitochondrial disease", Ann Neurol, 77, pp.753~759 (2015)
4. Anderson S, "Sequence and organization of the human mitochondrial genome", Nature, 290, pp.457~465 (1981)
5. Chen X, "Rearranged mitochondrial genomes are present in human oocytes", Am J Hum Genet, 57, pp.239~247 (1995)
6. Taylor RW, "Mitochondrial DNA mutations in human disease", Nat Rev Genet, 6, pp.389~402 (2005)
7. Kopinski PK, "Mitochondrial DNA variation and cancer", Nat Rev Cancer, 21, pp.431~445 (2021)
8. Kogelnik AM, "MITOMAP : a human mitochondrial genome database", Nucleic Acids Res, 24, pp.177~179 (1996)
9. He Y, "Heteroplasmic mitochondrial DNA mutations in normal and tumour cells", Nature, 464, pp.610~614 (2010)
10. Rossignol R, "Mitochondrial threshold effects", Biochem J, 370, pp.751~762 (2003)
11. Jinek M, "A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity", Science, 337, pp.816~821 (2012)
12. Komor AC, "Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage", Nature, 533, pp.420~424 (2016)
13. Gaudelli NM, "Programmable base editing of A*T to G*C in genomic DNA without DNA cleavage", Nature, 551, pp.464~471 (2017)
14. Anzalone AV, "Search-and-replace genome editing without double-strand breaks or donor DNA", Nature, 576, pp.149~157 (2019)
15. Gammage PA, "Mitochondrial genome engineering : the revolution may not be CRISPR-Ized", Trends Genet, 34, pp.101~110 (2018)
16. Fontana GA, "Mechanisms of replication and repair in mitochondrial DNA deletion formation", Nucleic Acids Res, 48, pp.11244~11258 (2020)
17. Silva-Pinheiro P, "The potential of mitochondrial genome engineering", Nat Rev Genet, 23, pp.199~214 (2022)
18. Peeva V, "Linear mitochondrial DNA is rapidly degraded by components of the replication machinery", Nat Commun, 9, pp.1727~ (2018)
19. Krokan HE, "Base excision repair", Cold Spring Harb Perspect Biol, 5, pp.a012583~ (2013)
20. Omura T, "Mitochondria-targeting sequence, a multi-role sorting sequence recognized at all steps of protein import into mitochondria", J Biochem, 123, pp.1010~1016 (1998)
21. Gammage PA, "Mitochondrially targeted ZFNs for selective degradation of pathogenic mitochondrial genomes bearing large-scale deletions or point mutations", EMBO Mol Med, 6, pp.458~466 (2014)
22. Gammage PA, "Nearcomplete elimination of mutant mtDNA by iterative or dynamic dose-controlled treatment with mtZFNs", Nucleic Acids Res, 44, pp.7804~7816 (2016)
23. Gammage PA, "Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo", Nat Med, 24, pp.1691~1695 (2018)
24. Reddy P, "Selective elimination of mitochondrial mutations in the germline by genome editing", Cell, 161, pp.459~469 (2015)
25. Bacman SR, "Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs", Nat Med, 19, pp.1111~1113 (2013)
26. Hashimoto M, "MitoTALEN : a general approach to reduce mutant mtDNA loads and restore oxidative phosphorylation function in mitochondrial diseases", Mol Ther, 23, pp.1592~1599 (2015)
27. Bacman SR, "MitoTALEN reduces mutant mtDNA load and restores tRNA(Ala)levels in a mouse model of heteroplasmic mtDNA mutation", Nat Med, 24, pp.1696~1700 (2018)
28. Yang Y, "Targeted elimination of mutant mitochondrial DNA in MELAS-iPSCs by mito-TALENs", Protein Cell, 9, pp.283~297 (2018)
29. Yahata N, "Elimination of mutant mtDNA by an optimized mpTALEN restores differentiation capacities of heteroplasmic MELAS-iPSCs", Mol Ther Methods Clin Dev, 20, pp.54~68 (2021)
30. Srivastava S, "Manipulating mitochondrial DNA heteroplasmy by a mitochondrially targeted restriction endonuclease", Hum Mol Genet, 10, pp.3093~3099 (2001)
31. Bayona-Bafaluy MP, "Rapid directional shift of mitochondrial DNA heteroplasmy in animal tissues by a mitochondrially targeted restriction endonuclease", Proc Natl Acad Sci U S A, 102, pp.14392~14397 (2005)
32. Bacman SR, "Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease", Gene Ther, 19, pp.1101~1106 (2012)
33. Tanaka M, "Gene therapy for mitochondrial disease by delivering restriction endonuclease SmaI into mitochondria", J Biomed Sci, 9, pp.534~541 (2002)
34. Alexeyev MF, "Selective elimination of mutant mitochondrial genomes as therapeutic strategy for the treatment of NARP and MILS syndromes", Gene Ther, 15, pp.516~523 (2008)
35. Pereira CV, "mitoTev-TALE : a monomeric DNA editing enzyme to reduce mu-tant mitochondrial DNA levels", EMBO Mol Med, 10, pp.e8084~ (2018)
36. Zekonyte U, "Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo", Nat Commun, 12, pp.3210~ (2021)
37. Minczuk M, "Development of a single-chain, quasidimeric zinc-finger nuclease for the selective degradation of mutated human mitochondrial DNA", Nucleic Acids Res, 36, pp.3926~3938 (2008)
38. Mok BY, "A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing", Nature, 583, pp.631~637 (2020)
39. Cho SI, "Targeted A-to-G base editing in human mitochondrial DNA with programmable deaminases", Cell, 185, pp.1764~1776 (2022)
40. Richter MF, "Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity", Nat Biotechnol, 38, pp.883~891 (2020)
41. Yin L, "Structural basis of sequence-specific cytosine deamination by double-stranded DNA deaminase toxin DddA", Nat Struct Mol Biol, 30, pp.1153~1159 (2023)
42. Mok BY, "CRISPR-free base editors with enhanced activity and expanded targeting scope in mitochondrial and nuclear DNA", Nat Biotechnol, 40, pp.1378~1387 (2022)
43. Mi L, "DddA homolog search and engineering expand sequence compatibility of mitochondrial base editing", Nat Commun, 14, pp.874~ (2023)
44. Sun H, "Developing mitochondrial base editors with diverse context compatibility and high fidelity via saturated spacer library", Nat Commun, 14, pp.6625~ (2023)
45. Cheng K, "Engineering RsDddA as mitochondrial base editor with wide target compatibility and enhanced activity", Mol Ther Nucleic Acids, 34, pp.102028~ (2023)
46. Guo J, "A DddA ortholog-based and transactivator-assisted nuclear and mitochondrial cytosine base editors with expanded target compatibility", Mol Cell, 83, pp.1710~1724 (2023)
47. Huang J, "Discovery of deaminase functions by structure-based protein clustering", Cell, 186, pp.3182~3195 (2023)
48. Lee S, "Enhanced mitochondrial DNA editing in mice using nuclear-exported TALE-linked deaminases and nucleases", Genome Biol, 23, pp.211~ (2022)
49. Lim K, "Nuclear and mitochondrial DNA editing in human cells with zinc finger deaminases", Nat Commun, 13, pp.366~ (2022)
50. Willis JCW, "Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivo", Nat Commun, 13, pp.7204~ (2022)
51. Mok YG, "Base editing in human cells with monomeric DddA-TALE fusion deami-nases", Nat Commun, 13, pp.4038~ (2022)
52. Wei Y, "Mitochondrial base editor DdCBE causes substantial DNA off-target editing in nuclear genome of embryos", Cell Discov, 8, pp.27~ (2022)
53. Lei Z, "Mitochondrial base editor induces substantial nuclear off-target mutations", Nature, 606, pp.804~811 (2022)
54. Lee S, "Precision mitochondrial DNA editing with high-fidelity DddA-derived base editors", Nat Biotechnol, 41, pp.378~386 (2023)
55. Guo J, "DdCBE mediates efficient and inheritable modifications in mouse mitochondrial genome", Mol Ther Nucleic Acids, 27, pp.73~80 (2022)
56. Chen X, "DdCBE-mediated mitochondrial base editing in human 3PN embryos", Cell Discov, 8, pp.8~ (2022)
57. Yi Z, "Strand-selective base editing of human mitochondrial DNA using mitoBEs", Nat Biotechnol, pp.1~12 (2023)
58. Hu J, "Strand-preferred base editing of organellar and nuclear genomes using CyDENT", Nat Biotechnol, pp.1~10 (2023)
59. Paunovska K, "Drug delivery systems for RNA therapeutics", Nat Rev Genet, 23, pp.265~280 (2022)
60. Degors IMS, "Carriers break barriers in drug delivery : endocytosis and endosomal escape of gene delivery vectors", Acc Chem Res, 52, pp.1750~1760 (2019)
61. Lee H, "Mitochondrial DNA editing in mice with DddA-TALE fusion deaminases", Nat Commun, 12, pp.1190~ (2021)
62. Silva-Pinheiro P, "A library of base editors for the precise ablation of all protein-coding genes in the mouse mitochondrial genome", Nat Biomed Eng, 7, pp.692~703 (2023)
63. Guo J, "Precision modeling of mitochondrial diseases in zebrafish via DdCBE-mediated mtDNA base editing", Cell Discov, 7, pp.78~ (2021)
64. Sabharwal A, "The FusX TALE Base Editor(FusXTBE)for rapid mitochondrial DNA programming of human cells in vitro and zebrafish disease models in vivo", CRISPR J, 4, pp.799~821 (2021)
65. Qi X, "Precision modeling of mitochondrial disease in rats via DdCBE-mediated mtDNA editing", Cell Discov, 7, pp.95~ (2021)
66. Qi X, "Expanding DdCBEmediated targeting scope to aC motif preference in rat", Mol Ther Nucleic Acids, 32, pp.1~12 (2023)
67. Tan L, "A conditional knockout rat resource of mitochondrial protein-coding genes via a DdCBE-induced premature stop codon", Sci Adv, 9, pp.2695~ (2023)
68. Wei Y, "Human cleaving embryos enable efficient mitochondrial base-editing with DdCBE", Cell Discov, 8, pp.7~ (2022)
69. Wang D, "Adeno-associated virus vector as a platform for gene therapy delivery", Nat Rev Drug Discov, 18, pp.358~378 (2019)
70. Silva-Pinheiro P, "In vivo mitochondrial base editing via adeno-associated viral delivery to mouse postmitotic tissue", Nat Commun, 13, pp.750~ (2022)
71. Lek A, "Death after High-Dose rAAV9 gene therapy in a patient with Duchenne’s Muscular Dystrophy", N Engl J Med, 389, pp.1203~1210 (2023)
72. Kang BC, "Chloroplast and mitochondrial DNA editing in plants", Nat Plants, 7, pp.899~905 (2021)

관련 논문AI추천