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 | 137 | 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 | 19 | 2023 |
In vivo multiplex gene targeting with Streptococcus pyogens and Campylobacter jejuni Cas9 for pancreatic cancer modeling in wild-type animal YJ Chang, J Bae, Y Zhao, G Lee, J Han, YH Lee, OJ Koo, S Seo, YK Choi, ... Journal of Veterinary Science 21 (2), e26, 2020 | 17 | 2020 |
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 |
High homology-directed repair using mitosis phase and nucleus localizing signal JP Han, YJ Chang, DW Song, BS Choi, OJ Koo, SY Yi, TS Park, SC Yeom International journal of molecular sciences 21 (11), 3747, 2020 | 11 | 2020 |
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 | 8 | 2023 |
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 | 7 | 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 LNP-CRISPR approaches for the treatment of hemophilia JH Lee, JP Han Molecular Diagnosis & Therapy 28 (3), 239-248, 2024 | 5 | 2024 |
The position of the target site for engineered nucleases improves the aberrant mRNA clearance in in vivo genome editing JH Lee, S Yu, TW Nam, J Roh, Y Jin, JP Han, JY Cha, YK Kim, SC Yeom, ... Scientific reports 10 (1), 4173, 2020 | 3 | 2020 |
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 |
Polyphenol-Enabled 2D Nanopatch for Enhanced Nasal Mucoadhesion and Immune Activation JP Han, YR Nam, HY Chung, H Lee, SC Yeom Nano Letters 24 (33), 10380-10387, 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 |
Mitosis stage is optimal timing for CRISPR ribonucleoprotein mediated single strand template repair JP Han, SC Yeom 한국실험동물학회 학술발표대회 논문집, 139-139, 2019 | | 2019 |