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
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2024.12.020
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