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

DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome.

DNA-PK 抑制剂可提高 HSPC 中基因编辑的效率,从而利用 CRISPR 技术治疗 X 连锁高 IgM 综合征

Pugliano Cole M, Berger Mason, Ray Roslyn M, Sapkos Kai, Wu Betty, Laird Aidan, Ye Yidian, Thomson Daniel, DeGottardi M Quinn, Khan Iram F, Tatiossian Kristina, Miles Brodie A, Aeschimann Florian, Pasquier Jerome, Kim Mihee M, Rawlings David J

TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

利用TALEN介导的基因编辑技术对人类造血干细胞中的HBG基因进行编辑,可诱导治疗性胎儿血红蛋白的产生。

Lux, Christopher T; Pattabhi, Sowmya; Berger, Mason; Nourigat, Cynthia; Flowers, David A; Negre, Olivier; Humbert, Olivier; Yang, Julia G; Lee, Calvin; Jacoby, Kyle; Bernstein, Irwin; Kiem, Hans-Peter; Scharenberg, Andrew; Rawlings, David J