日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Estradiol rescues male hydroxyl radical-mediated Charcot-Marie-Tooth 2Z by Morc2a stabilization through autophagy inhibition in a murine model.

在小鼠模型中,雌二醇通过抑制自噬来稳定 Morc2a,从而挽救雄性羟自由基介导的 Charcot-Marie-Tooth 2Z 病

Kim Ji Won, Nam Soo Hyun, Lee Geon Seong, Chung Hye Yoon, Kim Eun Young, Han Jeong Pil, Jang Jae-Hyung, Choi Byung-Ok, Yeom Su Cheong

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

利用病毒和非病毒载体,通过基因重平衡和治疗性基因敲入相结合的方法,进行体内基因组编辑以治疗B型血友病。

Lee, Jeong Hyeon; Han, Jeong Pil; Song, Dong Woo; Lee, Geon Seong; Choi, Beom Seok; Kim, MinJeong; Lee, Yeji; Kim, Seokjoong; Lee, Hyukjin; Yeom, Su Cheong

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

利用244-cis LNP和低剂量AAV进行体内基因组编辑,在A型血友病小鼠中达到了治疗阈值。

Han, Jeong Pil; Lee, Yeji; Lee, Jeong Hyeon; Chung, Hye Yoon; Lee, Geon Seong; Nam, Yu Ri; Choi, Myeongjin; Moon, Kyoung-Sik; Lee, Haeshin; Lee, Hyukjin; Yeom, Su Cheong

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

利用脂质纳米颗粒将 CRISPR-Cas9 递送至体内,可实现抗凝血酶基因编辑,从而为血友病 A 和 B 的可持续治疗提供可能。

Jeong Pil Han ,MinJeong Kim ,Beom Seok Choi ,Jeong Hyeon Lee ,Geon Seong Lee ,Michaela Jeong ,Yeji Lee ,Eun-Ah Kim ,Hye-Kyung Oh ,Nanyeong Go ,Hyerim Lee ,Kyu Jun Lee ,Un Gi Kim ,Jae Young Lee ,Seokjoong Kim ,Jun Chang ,Hyukjin Lee ,Dong Woo Song ,Su Cheong Yeom

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

基因组编辑介导的治疗基因敲入改善了血友病模型中的疾病表型

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

Novel Severe Hemophilia A Mouse Model with Factor VIII Intron 22 Inversion

具有第 VIII 因子内含子 22 倒位的新型严重血友病 A 小鼠模型

Jeong Pil Han, Dong Woo Song, Jeong Hyeon Lee, Geon Seong Lee, Su Cheong Yeom