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[국내논문] 유전자 교정 기술의 생의학적 응용
Biomedical Application of Gene Editing 원문보기

産業技術硏究 : 江原大學校 産業技術硏究所 = Journal of industrial technology, v.42 no.1, 2022년, pp.29 - 36  

박주찬 (College of Pharmacy, Seoul National University) ,  장현기 (Division of Chemical Engineering and Bioengineering, College of Art Culture and Engineering)

Abstract AI-Helper 아이콘AI-Helper

The CRISPR system has revolutionized gene editing field. Cas9-mediated gene editing such as Indel induction or HDR enable targeted gene disruption or precise correction of mutation. Moreover, CRISPR-based new editing tools have been developed such as base editors. In this review, we focus on gene ed...

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표/그림 (4)

참고문헌 (45)

  1. Hsu, Patrick D., Lander, Eric S.,Zhang, F., 2014, Development and?Applications of CRISPR-Cas9 for Genome Engineering, Cell 157:6?1262-1278. 

  2. Jang, H. K., Song, B., Hwang, G. H.,Bae, S., 2020, Current trends?in gene recovery mediated by the CRISPR-Cas system, Exp Mol?Med 52:7 1016-1027. 

  3. Ihry, R. J., Worringer, K. A., Salick, M. R., Frias, E., Ho, D.,?Theriault, K., Kommineni, S., Chen, J., Sondey, M., Ye, C.,?Randhawa, R., Kulkarni, T., Yang, Z., McAllister, G., Russ, C.,?Reece-Hoyes, J., Forrester, W., Hoffman, G. R., Dolmetsch,?R.,Kaykas, A., 2018, p53 inhibits CRISPR-Cas9 engineering in?human pluripotent stem cells, Nat Med 24:7 939-946. 

  4. Haapaniemi, E., Botla, S., Persson, J., Schmierer, B.,Taipale, J.,?2018, CRISPR-Cas9 genome editing induces a p53-mediated DNA?damage response, Nat Med 24:7 927-930. 

  5. Shin, H. Y., Wang, C., Lee, H. K., Yoo, K. H., Zeng, X., Kuhns,?T., Yang, C. M., Mohr, T., Liu, C.,Hennighausen, L., 2017,?CRISPR/Cas9 targeting events cause complex deletions and?insertions at 17 sites in the mouse genome, Nat Commun 8 15464. 

  6. Kosicki, M., Tomberg, K.,Bradley, A., 2018, Repair of?double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements, Nat Biotechnol 36:8 765-771. 

  7. Komor, A. C., Kim, Y. B., Packer, M. S., Zuris, J. A.,Liu, D. R.,?2016, Programmable editing of a target base in genomic DNA?without double-stranded DNA cleavage, Nature 533:7603 420-424. 

  8. Gaudelli, N. M., Komor, A. C., Rees, H. A., Packer, M. S., Badran,?A. H., Bryson, D. I.,Liu, D. R., 2017, Programmable base editing?of AT to GC in genomic DNA without DNA cleavage, Nature?551:7681 464-471. 

  9. Dutta, D., Heo, I.,Clevers, H., 2017, Disease Modeling in Stem?Cell-Derived 3D Organoid Systems, Trends Mol Med 23:5 393-410. 

  10. Yamanaka, S., 2020, Pluripotent Stem Cell-Based Cell?Therapy-Promise and Challenges, Cell Stem Cell 27:4 523-531. 

  11. Rowe, R. G.,Daley, G. Q., 2019, Induced pluripotent stem cells in?disease modelling and drug discovery, Nat Rev Genet 20:7 377-388. 

  12. Wang, H. X., Li, M., Lee, C. M., Chakraborty, S., Kim, H. W.,?Bao, G.,Leong, K. W., 2017, CRISPR/Cas9-Based Genome Editing?for Disease Modeling and Therapy: Challenges and Opportunities?for Nonviral Delivery, Chem Rev 117:15 9874-9906. 

  13. Sebastiano, V., Zhen, H. H., Haddad, B., Bashkirova, E., Melo, S.?P., Wang, P., Leung, T. L., Siprashvili, Z., Tichy, A., Li, J., Ameen,?M., Hawkins, J., Lee, S., Li, L., Schwertschkow, A., Bauer, G.,?Lisowski, L., Kay, M. A., Kim, S. K., Lane, A. T., Wernig, M.,Oro,?A. E., 2014, Human COL7A1-corrected induced pluripotent stem?cells for the treatment of recessive dystrophic epidermolysis bullosa,?Sci Transl Med 6:264 264ra163. 

  14. Niu, X., He, W., Song, B., Ou, Z., Fan, D., Chen, Y., Fan, Y.,Sun,?X., 2016, Combining Single Strand Oligodeoxynucleotides and?CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced?Pluripotent Stem Cells, J Biol Chem 291:32 16576-16585. 

  15. Ortiz-Virumbrales, M., Moreno, C. L., Kruglikov, I., Marazuela, P.,?Sproul, A., Jacob, S., Zimmer, M., Paull, D., Zhang, B., Schadt,?E. E., Ehrlich, M. E., Tanzi, R. E., Arancio, O., Noggle, S.,Gandy,?S., 2017, CRISPR/Cas9-Correctable mutation-related molecular and?physiological phenotypes in iPSC-derived Alzheimer's PSEN2?(N141I) neurons, Acta Neuropathol Commun 5:1 77. 

  16. Steyer, B., Bu, Q., Cory, E., Jiang, K., Duong, S., Sinha, D.,?Steltzer, S., Gamm, D., Chang, Q.,Saha, K., 2018, Scarless Genome?Editing of Human Pluripotent Stem Cells via Transient Puromycin?Selection, Stem Cell Reports 10:2 642-654. 

  17. Li, X. L., Li, G. H., Fu, J., Fu, Y. W., Zhang, L., Chen, W.,?Arakaki, C., Zhang, J. P., Wen, W., Zhao, M., Chen, W. V.,?Botimer, G. D., Baylink, D., Aranda, L., Choi, H., Bechar, R.,?Talbot, P., Sun, C. K., Cheng, T.,Zhang, X. B., 2018, Highly?efficient genome editing via CRISPR-Cas9 in human pluripotent?stem cells is achieved by transient BCL-XL overexpression,?Nucleic Acids Res 46:19 10195-10215. 

  18. Shahryari, A., Moya, N., Siehler, J., Wang, X., Burtscher,?I.,Lickert, H., 2021, Increasing Gene Editing Efficiency for?CRISPR-Cas9 by Small RNAs in Pluripotent Stem Cells, Crispr?j 4:4 491-501. 

  19. Howden, S. E., McColl, B., Glaser, A., Vadolas, J., Petrou, S.,?Little, M. H., Elefanty, A. G.,Stanley, E. G., 2016, A Cas9 Variant?for Efficient Generation of Indel-Free Knockin or Gene-Corrected?Human Pluripotent Stem Cells, Stem Cell Reports 7:3 508-517. 

  20. Yang, D., Scavuzzo, M. A., Chmielowiec, J., Sharp, R., Bajic,?A.,Borowiak, M., 2016, Enrichment of G2/M cell cycle phase in?human pluripotent stem cells enhances HDR-mediated gene repair?with customizable endonucleases, Sci Rep 6 21264. 

  21. Riesenberg, S.,Maricic, T., 2018, Targeting repair pathways with?small molecules increases precise genome editing in pluripotent?stem cells, Nat Commun 9:1 2164. 

  22. Martin, R. M., Ikeda, K., Cromer, M. K., Uchida, N., Nishimura,?T., Romano, R., Tong, A. J., Lemgart, V. T., Camarena, J.,?Pavel-Dinu, M., Sindhu, C., Wiebking, V., Vaidyanathan, S.,?Dever, D. P., Bak, R. O., Laustsen, A., Lesch, B. J., Jakobsen,?M. R., Sebastiano, V., Nakauchi, H.,Porteus, M. H., 2019, Highly?Efficient and Marker-free Genome Editing of Human Pluripotent?Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated?Homologous Recombination, Cell Stem Cell 24:5 821-828.e825. 

  23. Kim, K. T., Park, J. C., Jang, H. K., Lee, H., Park, S., Kim, J.,?Kwon, O. S., Go, Y. H., Jin, Y., Kim, W., Lee, J., Bae, S.,Cha,?H. J., 2020, Safe scarless cassette-free selection of genome-edited?human pluripotent stem cells using temporary drug resistance,?Biomaterials 262 120295. 

  24. Lee, M. O., Moon, S. H., Jeong, H. C., Yi, J. Y., Lee, T. H.,?Shim, S. H., Rhee, Y. H., Lee, S. H., Oh, S. J., Lee, M. Y., Han,?M. J., Cho, Y. S., Chung, H. M., Kim, K. S.,Cha, H. J., 2013,?Inhibition of pluripotent stem cell-derived teratoma formation by?small molecules, Proc Natl Acad Sci U S A 110:35 E3281-3290. 

  25. Kim, K. T., Jeong, H. C., Kim, C. Y., Kim, E. Y., Heo, S. H., Cho, S. J., Hong, K. S.,Cha, H. J., 2017, Intact wound repair activity?of human mesenchymal stem cells after YM155 mediated selective?ablation of undifferentiated human embryonic stem cells, J Dermatol?Sci 86:2 123-131. 

  26. Rees, H. A.,Liu, D. R., 2018, Base editing: precision chemistry on?the genome and transcriptome ofliving cells, Nature Reviews?Genetics 19:12 770-788. 

  27. Song, M., Kim, H. K., Lee, S., Kim, Y., Seo, S.-Y., Park, J., Choi,?J. W., Jang, H., Shin, J. H., Min, S., Quan, Z., Kim, J. H., Kang,?H. C., Yoon, S.,Kim, H. H., 2020, Sequence-specific prediction of?the efficiencies of adenine and cytosine base editors, Nature?Biotechnology 38:9 1037-1043. 

  28. Park, J. C., Jang, H. K., Kim, J., Han, J. H., Jung, Y., Kim, K.,?Bae, S.,Cha, H. J., 2022, High expression of uracil DNA glycosylase?determines C to T substitution in human pluripotent stem cells, Mol?Ther Nucleic Acids 27 175-183. 

  29. Komor, A. C., Zhao, K. T., Packer, M. S., Gaudelli, N. M.,?Waterbury, A. L., Koblan, L. W., Kim, Y. B., Badran, A. H.,Liu,?D. R., 2017, 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 3:8 eaao4774. 

  30. Park, J. C., Kim, J., Jang, H. K., Lee, S. Y., Kim, K. T., Kwon,?E. J., Park, S., Lee, H. S., Choi, H., Park, S. Y., Choi, H. J., Park,?S. J., Moon, S. H., Bae, S.,Cha, H. J., 2022, Multiple isogenic?GNE-myopathy modeling with mutation specific phenotypes from?human pluripotent stem cells by base editors, Biomaterials 282?121419. 

  31. Truong, D. J., Kuhner, K., Kuhn, R., Werfel, S., Engelhardt, S.,?Wurst, W.,Ortiz, O., 2015, Development of an intein-mediated?split-Cas9 system for gene therapy, Nucleic Acids Res 43:13?6450-6458. 

  32. Chen, Y., Zhi, S., Liu, W., Wen, J., Hu, S., Cao, T., Sun, H., Li,?Y., Huang, L., Liu, Y., Liang, P.,Huang, J., 2020, Development of?Highly Efficient Dual-AAV Split Adenosine Base Editor for In Vivo?Gene Therapy, Small Methods 4:9 2000309. 

  33. Keeler, A. M.,Flotte, T. R., 2019, Recombinant Adeno-Associated?Virus Gene Therapy in Light of Luxturna (and Zolgensma and?Glybera): Where Are We, and How Did We Get Here?, Annu Rev?Virol 6:1 601-621. 

  34. Jo, D. H., Jang, H.-K., Cho, C. S., Han, J. H., Ryu, G., Jung,?Y., Bae, S.,Kim, J. H., 2021, Therapeutic adenine base editing?corrects nonsense mutation and improves visual function in a?mouse model of Leber congenital amaurosis, bioRxiv?2021.2001.2007.425822. 

  35. Raguram, A., Banskota, S.,Liu, D. R., 2022, Therapeutic invivo?delivery of gene editing agents, Cell 185:15 2806-2827. 

  36. Jang, H. K., Jo, D. H., Lee, S. N., Cho, C. S., Jeong, Y. K., Jung,?Y., Yu, J., Kim, J. H., Woo, J. S.,Bae, S., 2021, High-purity?production and precise editing of DNA base editing?ribonucleoproteins, Sci Adv 7:35. 

  37. Nahmad, A. D., Reuveni, E., Goldschmidt, E., Tenne, T.,?Liberman, M., Horovitz-Fried, M., Khosravi, R., Kobo, H.,?Reinstein, E., Madi, A., Ben-David, U.,Barzel, A., 2022, Frequent?aneuploidy in primary human T cells after CRISPRCas9 cleavage,?Nature Biotechnology. 

  38. Slesarenko, Y. S., Lavrov, A. V.,Smirnikhina, S. A., 2022,?Off-target effects of base editors: what we know and how we can?reduce it, Curr Genet 68:1 39-48. 

  39. Richter, M. F., Zhao, K. T., Eton, E., Lapinaite, A., Newby, G.?A., Thuronyi, B. W., Wilson, C., Koblan, L. W., Zeng, J., Bauer,?D. E., Doudna, J. A.,Liu, D. R., 2020, Phage-assisted evolution?of an adenine base editor with improved Cas domain compatibility?and activity, Nature Biotechnology 38:7 883-891. 

  40. Li, S., Yuan, B., Cao, J., Chen, J., Chen, J., Qiu, J., Zhao, X.-M.,?Wang, X., Qiu, Z.,Cheng, T.-L., 2020, Docking sites inside Cas9?for adenine base editing diversification and RNA off-target?elimination, Nature Communications 11:1 5827. 

  41. Paunovska, K., Loughrey, D.,Dahlman, J. E., 2022, Drug delivery?systems for RNA therapeutics, Nature Reviews Genetics 23:5?265-280. 

  42. Musunuru, K., Chadwick, A. C., Mizoguchi, T., Garcia, S. P.,?DeNizio, J. E., Reiss, C. W., Wang, K., Iyer, S., Dutta, C.,?Clendaniel, V., Amaonye, M., Beach, A., Berth, K., Biswas, S.,?Braun, M. C., Chen, H.-M., Colace, T. V., Ganey, J. D.,?Gangopadhyay, S. A., Garrity, R., Kasiewicz, L. N., Lavoie, J.,?Madsen, J. A., Matsumoto, Y., Mazzola, A. M., Nasrullah, Y. S.,?Nneji, J., Ren, H., Sanjeev, A., Shay, M., Stahley, M. R., Fan,?S. H. Y., Tam, Y. K., Gaudelli, N. M., Ciaramella, G., Stolz, L.?E., Malyala, P., Cheng, C. J., Rajeev, K. G., Rohde, E., Bellinger,?A. M.,Kathiresan, S., 2021, In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates, Nature 593:7859?429-434. 

  43. Rothgangl, T., Dennis, M. K., Lin, P. J. C., Oka, R., Witzigmann,?D., Villiger, L., Qi, W., Hruzova, M., Kissling, L., Lenggenhager,?D., Borrelli, C., Egli, S., Frey, N., Bakker, N., Walker, J. A., Kadina,?A. P., Victorov, D. V., Pacesa, M., Kreutzer, S., Kontarakis, Z.,?Moor, A., Jinek, M., Weissman, D., Stoffel, M., van Boxtel, R.,?Holden, K., Pardi, N., Thony, B., Haberle, J., Tam, Y. K., Semple,?S. C.,Schwank, G., 2021, In vivo adenine base editing of PCSK9?in macaques reduces LDL cholesterol levels, Nature Biotechnology?39:8 949-957. 

  44. Yeh, W.-H., Chiang, H., Rees, H. A., Edge, A. S. B.,Liu, D. R.,?2018, In vivo base editing of post-mitotic sensory cells, Nature?Communications 9:1 2184. 

  45. Banskota, S., Raguram, A., Suh, S., Du, S. W., Davis, J. R., Choi,?E. H., Wang, X., Nielsen, S. C., Newby, G. A., Randolph, P. B.,?Osborn, M. J., Musunuru, K., Palczewski, K.,Liu, D. R., 2022,?Engineered virus-like particles for efficient invivo delivery of?therapeutic proteins, Cell 185:2 250-265.e216. 

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