Geon Seong Lee

In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

Jeong Pil Han, M. Kim, Beom Seok Choi et al. · Science Advances · 2022 · 185 citations · Full text

Life Sciences, Biotechnology, Hematology +17

In vivo genome editing for hemophilia B therapy by the combination of rebalancing and therapeutic gene knockin using a viral and non-viral vector

Jeong Hyeon Lee, Jeong Pil Han, Dong Woo Song et al. · Molecular Therapy — Nucleic Acids · 2023 · 39 citations · Full text

Medicine, Genetics, Hematology +17

Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia

Jeong Hyeon Lee, Hye-Kyung Oh, Beom Seok Choi et al. · Molecular Therapy — Nucleic Acids · 2022 · 17 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17

In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice

Jeong Pil Han, Yeji Lee, Jeong Hyeon Lee et al. · Molecular Therapy — Nucleic Acids · 2023 · 16 citations · Full text

Life Sciences, Genetics, Molecular Biology +12

<i>Morc2a</i>p.S87L mutant mice develop peripheral and central neuropathies associated with neuronal DNA damage and apoptosis

Geon Seong Lee, Geon Kwak, Ji Hyun Bae et al. · Disease Models & Mechanisms · 2021 · 12 citations · Full text

Medicine, Genetics, Neurology +17