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

Engraftment and persistence of HBB base-edited hematopoietic stem cells in nonhuman primates

HBB碱基编辑的造血干细胞在非人灵长类动物中的植入和持久性

Radtke, Stefan; Fields, Emily; Swing, Kyle; Kanestrom, Greta; Yen, Jonathan S; Pande, Dnyanada; Enstrom, Mark R; Humbert, Olivier; Weiss, Mitchell J; Liu, David R; Newby, Gregory A; Kiem, Hans-Peter

Preclinical development of lentiviral vector gene therapy for Diamond-Blackfan anemia syndrome.

针对Diamond-Blackfan贫血综合征的慢病毒载体基因疗法的临床前开发

Bhoopalan Senthil Velan, Mayuranathan Thiyagaraj, Liu Nana, Mayberry Kalin, Yao Yu, Zhang Jingjing, Métais Jean-Yves, Yan Koon-Kiu, Throm Robert E, Ellis Steven R, Ju Yan, Han Lei, Suryaprakash Shruthi, Palmer Lance E, Zhou Sheng, Yu Jiyang, Cheng Yong, Yen Jonathan S, Gottschalk Stephen, Weiss Mitchell J

A mouse model for hemoglobin SC disease recapitulates characteristic human pathologies

小鼠血红蛋白SC疾病模型重现了人类的特征性病理。

Zhai, Jinbin; Papizan, James B; Yao, Yu; Arnold, Elizabeth D; Yu, Jing; Tran, Tam; Geiger, Natalie; Gollamudi, Jahnavi; Little, Danielle R; Jadeja, Ravirajsinh N; John, Jaison; Khalighifar, Ali; Mayberry, Kalin; McEvoy, Justina; Rooney, Randi; Venkataraman, Krishnan; Vu, Hieu S; Zhang, Jingjing; Dyer, Michael; Gallagher, Patrick G; Hyacinth, Hyacinth I; Janke, Laura J; Martin, Pamela M; Ni, Min; Xu, Jian; Yen, Jonathan S; Zheng, Yan; Weiss, Mitchell J; Pruett-Miller, Shondra M

BCL11A-deficient human erythropoiesis is impaired in vitro and after xenotransplantation into mice.

BCL11A 缺陷的人类红细胞生成在体外和异种移植到小鼠后均受到损害

Jang Yoonjeong, Feng Ruopeng, Palmer Lance E, Mayuranathan Thiyagaraj, Yao Yu, Mayberry Kalin, Zhou Sheng, Xu Jian, Gossett Jeffrey M, Kang Guolian, Cheng Yong, Yen Jonathan S, Weiss Mitchell J