Gene Therapy Laboratory
Pharmaceutical Science at Kyung Hee University
RECENT
PUBLICATIONS
1. Gee MS, Kwon E, Song MH, Jeon SH, Kim N, Lee JK, Koo T. CRISPR base editing-mediated correction of a tau mutation rescues cognitive decline in a mouse model of tauopathy. Transl Neurodegener. 2024 Apr 12;13(1):21.
2. Lee Y, Ju YJ, Gee MS, Jeon SH, Kim N, Koo T, Lee JK. Survivin enhances hippocampal neurogenesis and cognitive function in Alzheimer's disease mouse model. CNS Neurosci Ther. 2024 Apr;30(4):e14509.
3. Kweon J, Jang A, Kwon E, Kim U, Shin H, See J, Jang G, Lee C, Koo T, Kim S, Kim Y. Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery. Experimental & Molecular Medicine. 2023, Feb 1. https://doi.org/10.1038/s12276-023-00938-w
4. Choi E, Hwang H, Kwon E, Kim D, Koo T. Expanded targeting scope of LbCas12a variants allows editing of multiple oncogenic mutations. Molecular Therapy: Nucleic Acid. 2022 Dec; 30, 131-142. .
5. Choi E, Koo T. Muscular Dystrophy Therapy Using Viral Vector-based CRISPR/Cas. Biotechnologies for gene therapy. 2022 Jun 3; 61–83. https://link.springer.com/chapter/10.1007/978-3-030-93333-3_4 .
6. Do Yon Kim, Jeong Mi Lee, Su Bin Moon, Hyun Jung Chin, Seyeon Park, Youjung Lim, Daesik Kim , Koo T, Jeong-Heon Ko and Yong-Sam Kim Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus Nature Biotechnology. 2021 Sep 2; 40, 92-102.
7. Song M, Koo T. Recent advances in CRISPR technologies for genome editing. Arch. Pharm. Res. 2021 Jun 23; 44, 537–552.
8. Choi E, Koo T. CRISPR Technologies for the Treatment of Duchenne Muscular Dystrophy. Molecular Therapy. 2021 Apr 3:S1525-0016(21)00189-1.
9. Yoon AR, Jung BK, Choi E, Chung E, Hong JW, Kim JS, Koo T, Yun CO. CRISPR-Cas12a with an oAd Induces Precise and Cancer-Specific Genomic Reprogramming of EGFR and Efficient Tumor Regression. Molecular Therapy. 2020 Oct 7;28(10):2286-2296.
10. .Jo DH†, Koo T†, Cho CS, Kim JH, Kim JS, Kim JH. Long-term effects of in vivo genome editing in the mouse retina using Campylobacter jejuni Cas9 expressed via adeno-associated virus.. Molecular Therapy 2019, Jan 2;27(1);130-136. †equal contribution
11. SM Ryu†, Koo T†, K Kim†, K Lim†, G Back, ST Kim, HS Kim, D Kim, H Lee, E Chung, JS Kim. Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy. Nature Biotechnology 2018, Apr 27. 36(6); 536-539. †equal contribution
12. Koo T†, Park SW†, Kim D,KimJH,Cho HY,Kim JH,Kim JS. CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration. Nature Communications 2018, May 10. 10;9(1):1855. †equal contribution
13. Koo T, Lu-Nguyen N, Kim EJ, Malerba A, Kim D, Cappellari O, Cho HY, Dickson G, Popplewell L, Kim JS. Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9. Molecular Therapy 2018, Mar 30. pii: S1525-0016(18)30156-4.
14. Kim SJ†, Koo T†, Gee HG†, Cho HY, LEE GL, Kim JS. CRISPR RNAs trigger innate immune responses in human cells. Genome Research 2018 Feb 22. †equal contribution
15. Koo T† ,Yoon AR†, Cho HY, Bae S, Yun CO, Kim JS, Selective disruption of an oncogenic mutant allele by CRISPR/Cas9 induces efficient tumor regression. Nucleic Acids Research 2017 Jul 27;45(13):7897-7908. †equal contribution
16. Kim E†, Koo T†, Park SW†, Kim D,Kim K,Cho HY,Song DW,Lee KJ,Jung MH,Kim S,Kim JH,Kim JH,Kim JS. In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni. Nature Communications 2017 Feb 21;8:14500. †equal contribution
17. C Guiner , L Servais , M Montus , T Larcher , B Fraysse , S Moullec , M Allais , V François , M Dutilleul , T Koo , A Malerba , JL Thibaut , B Matot , M Devaux , J Le Duff , J Deschamps , I Barthelemy , S Blot , I Testault , K Wahbi , S Ederhy , S Martin , P Veron , C Georger , T Athanasopoulos , C Masurier , F Mingozzi , P Carlier , B Gjata , J Hogrel , O Adjali , F Mavilio , T Voit , M Philippe, G Dickson. Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy Nature Communications 2017 Jul 25;8:16105
18. Kim K,Park SW,Kim JH,Lee SH,Kim D,Koo T, Kim KE,Kim JH,Kim JS. Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration. Genome Research 2017 Mar;27(3):419-426
19. Koo T, Kim JS. Therapeutic applications of CRISPR RNA-guided genome editing. Briefings in Functional Genomics, Oxford University Press, 2017 Jan;16(1):38-45
20. Koo T†, Lee J†, Kim JS. Measuring and Reducing Off-Target Activities of Programmable Nucleases Including CRISPR-Cas9. Molecules and Cells. 2015 Jun;38(6):475-8. †equal contribution
21. Ezzat K,Aoki Y,Koo T, McClorey G,Benner L,Coenen-Stass A,O'Donovan L,Lehto T,Garcia-Guerra A,Nordin J,Saleh AF,Behlke M,Morris J,Goyenvalle A,Dugovic B,Leumann C,Gordon S,Gait MJ,El-Andaloussi S,Wood MJ.Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides. Nano Letters 2015 Jul 8;15(7):4364-73.
22. Rau F,Lainé J,Ramanoudjame L,Ferry A,Arandel L,Delalande O,Jollet A,Dingli F,Lee KY,Peccate C,Lorain S,Kabashi E,Athanasopoulos T,Koo T,Loew D,Swanson MS,Le Rumeur E,Dickson G,Allamand V,Marie J,Furling D. Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy. Nature Communications 2015 May 28;6:7205
23. JS Kim, T Koo, KM Kim, DS Kim, JE Kim, H Kim, H Hur. Composition for Genome Editing Comprising Cpf1 and Use thereof. Application: WO2017099494A8 2017-08-10.
24. T Koo, JS Kim A method for regulation of gene expression by expressing CAS9 protein from the two independent vector Application:WO2016080795A1 2016-05-26
25. Gait MJ, Arzumanov AA, Saleh AF, Wood MJ, Betts C, Koo T. Cell- penetrating peptides having a central hydrophobic domain. Grant: US9302014B2. 2016-04-5.