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

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

Ancestral β-globin gene haplotypes modify β-thalassemia severity in a mouse model

祖先β-珠蛋白基因单倍型会影响小鼠模型中β-地中海贫血的严重程度

Lechauve, Christophe; Keith, Julia; Dudley, Michael W; Fernandez, Alfonso G; Khandros, Eugene; Mayberry, Kalin; Mayuranathan, Thiyagaraj; Palmer, Lance; Qiu, Xiaohui; Sheppard, Heather; Telange, Rahul; Herz, Hans-Martin; Weiss, Mitchell J

The autophagy-activating kinase ULK1 mediates clearance of free α-globin in β-thalassemia

自噬激活激酶ULK1介导β-地中海贫血中游离α-珠蛋白的清除。

Lechauve, Christophe; Keith, Julia; Khandros, Eugene; Fowler, Stephanie; Mayberry, Kalin; Freiwan, Abdullah; Thom, Christopher S; Delbini, Paola; Romero, Emilio Boada; Zhang, Jingjing; Motta, Irene; Tillman, Heather; Cappellini, M Domenica; Kundu, Mondira; Weiss, Mitchell J