| In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy JP Han, MJ Kim, BS Choi, JH Lee, GS Lee, M Jeong, Y Lee, EA Kim, ... Science advances 8 (3), eabj6901, 2022 | 108 | 2022 |
| In vivo genome editing for hemophilia B therapy by the combination of rebalancing and therapeutic gene knockin using a viral and non-viral vector JH Lee, JP Han, DW Song, GS Lee, BS Choi, MJ Kim, Y Lee, S Kim, ... Molecular Therapy-Nucleic Acids 32, 161-172, 2023 | 14 | 2023 |
| Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia JH Lee, HK Oh, BS Choi, HH Lee, KJ Lee, UG Kim, J Lee, H Lee, GS Lee, ... Molecular Therapy-Nucleic Acids 29, 551-562, 2022 | 11 | 2022 |
| Positive correlation between nNOS and stress-activated bowel motility is confirmed by in vivo HiBiT system JP Han, JH Lee, GS Lee, OJ Koo, SC Yeom Cells 10 (5), 1028, 2021 | 8 | 2021 |
| Morc2a p.S87L mutant mice develop peripheral and central neuropathies associated with neuronal DNA damage and apoptosis GS Lee, G Kwak, JH Bae, JP Han, SH Nam, JH Lee, S Song, GD Kim, ... Disease Models & Mechanisms 14 (10), dmm049123, 2021 | 6 | 2021 |
| Novel severe hemophilia a mouse model with factor VIII intron 22 inversion JP Han, DW Song, JH Lee, GS Lee, SC Yeom Biology 10 (8), 704, 2021 | 6 | 2021 |
| In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice JP Han, Y Lee, JH Lee, HY Chung, GS Lee, YR Nam, M Choi, KS Moon, ... Molecular Therapy-Nucleic Acids 34, 2023 | 4 | 2023 |
| Morc2a variants cause hydroxyl radical-mediated neuropathy and are rescued by restoring GHKL ATPase HY Chung, GS Lee, SH Nam, JH Lee, JP Han, S Song, GD Kim, C Jung, ... Brain 147 (6), 2114-2127, 2024 | 2 | 2024 |
| In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia JH Lee, JP Han Molecular Diagnosis & Therapy 28 (3), 239-248, 2024 | 1 | 2024 |
| Comparison in vivo knock-in strategy using dual adeno associated virus JH Lee, DW Song, SJ Ahn, JP Han, GS Lee, SJ Kim, SC Yeom 한국실험동물학회 학술발표대회 논문집, 208-208, 2021 | | 2021 |
