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

Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies

通过对造血干细胞和祖细胞中的 HBG 启动子进行双重有益突变编辑,增强胎儿血红蛋白的生成,用于治疗 β-血红蛋白病

Prathibha Babu Chandraprabha #,Manoj Kumar K Azhagiri #,Vigneshwaran Venkatesan,Wendy Magis,Kirti Prasad,Sevanthy Suresh,Aswin Anand Pai,Srujan Marepally,Alok Srivastava,Kumarasamypet Murugesan Mohankumar,David I K Martin,Saravanabhavan Thangavel

High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation

在红细胞分化过程中,镰状突变的高水平校正在体内被放大

Wendy Magis, Mark A DeWitt, Stacia K Wyman, Jonathan T Vu, Seok-Jin Heo, Shirley J Shao, Finn Hennig, Zulema G Romero, Beatriz Campo-Fernandez, Suzanne Said, Matthew S McNeill, Garrett R Rettig, Yongming Sun, Yu Wang, Mark A Behlke, Donald B Kohn, Dario Boffelli, Mark C Walters, Jacob E Corn, David

CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells

利用 CRISPR-Cas9 技术对人红细胞中假定的胎儿珠蛋白抑制因子进行检测

Jennifer E Chung,Wendy Magis,Jonathan Vu,Seok-Jin Heo,Kirmo Wartiovaara,Mark C Walters,Ryo Kurita,Yukio Nakamura,Dario Boffelli,David I K Martin,Jacob E Corn,Mark A DeWitt