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

Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous disease

靶向基因编辑和近乎普遍的 CYBA 和 CYBB cDNA 插入作为慢性肉芽肿病的治疗方法

Wolff, Jonas Holst; Skov, Thomas Wisbech; Haslund, Didde; Dorset, Sofie Rahbek; Revenfeld, Anne Louise S; Aussel, Clotilde; Jørgensen, Sofie E; Holm, Mette; Thomsen, Martin K; Ammann, Sandra; Cathomen, Toni; Mogensen, Trine H; Møller, Bjarne Kuno; Bak, Rasmus O; Mikkelsen, Jacob Giehm

Gene editing in hematopoietic stem cells by co-delivery of Cas9/sgRNA ribonucleoprotein and templates for homology-directed repair in 'all-in-one' lentivirus-derived nanoparticles.

通过在“一体化”慢病毒衍生的纳米颗粒中共同递送 Cas9/sgRNA 核糖核蛋白和同源定向修复模板,对造血干细胞进行基因编辑

Andersen Sofie, Wolff Jonas Holst, Skov Thomas Wisbech, Janns Jacob Hørlück, Davis Luther John, Haldrup Jakob H, Haslund Didde, Revenfeld Anne Louise, Relkovic Dinko, Møller Bjarne K, Lund Jacob, Bak Rasmus O, Thomsen Emil Aagaard, Mikkelsen Jacob Giehm

Dominant-negative SERPING1 variants cause intracellular retention of C1 inhibitor in hereditary angioedema

显性负性SERPING1变异导致遗传性血管性水肿中C1抑制剂在细胞内滞留。

Haslund, Didde; Ryø, Laura Barrett; Seidelin Majidi, Sara; Rose, Iben; Skipper, Kristian Alsbjerg; Fryland, Tue; Bohn, Anja Bille; Koch, Claus; Thomsen, Martin K; Palarasah, Yaseelan; Corydon, Thomas J; Bygum, Anette; Nejsum, Lene N; Mikkelsen, Jacob Giehm