In vivo genome editing for hemophilia B therapy by the combination of rebalancing and therapeutic gene knockin using a viral and non-viral vector by Jeong Hyeon Lee, Jeong Pil Han, Dong Woo Song, Geon Seong Lee, Beom Seok Choi, MinJeong Kim, Yeji Lee, Seokjoong Kim, Hyukjin Lee, Su Cheong Yeom

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In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice by Jeong Pil Han, Yeji Lee, Jeong Hyeon Lee, Hye Yoon Chung, Geon Seong Lee, Yu Ri Nam, Myeongjin Choi, Kyoung-Sik Moon, Haeshin Lee, Hyukjin Lee, Su Cheong Yeom

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Morc2a p.S87L mutant mice develop peripheral and central neuropathies associated with neuronal DNA damage and apoptosis by Geon Seong Lee, Geon Kwak, Ji Hyun Bae, Jeong Pil Han, Soo Hyun Nam, Jeong Hyeon Lee, Sumin Song, Gap-Don Kim, Tae Sub Park, Yang Kyu Choi, Byung-Ok Choi, Su Cheong Yeom

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Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia by Jeong Hyeon Lee, Hye-Kyung Oh, Beom Seok Choi, Ho Hyeon Lee, Kyu Jun Lee, Un Gi Kim, Jina Lee, Hyerim Lee, Geon Seong Lee, Se Jun Ahn, Jeong Pil Han, Seokjoong Kim, Su Cheong Yeom, Dong Woo Song

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