Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy
利用靶向造血干细胞的脂质纳米颗粒构建致死性α-地中海贫血小鼠模型，并通过慢病毒基因治疗进行治疗。
期刊:Blood
影响因子:23.1
doi:10.1182/blood.2023023349
Chappell, Maxwell E; Breda, Laura; Tricoli, Lucas; Guerra, Amaliris; Jarocha, Danuta; Castruccio Castracani, Carlo; Papp, Tyler E; Tanaka, Naoto; Hamilton, Nolan; Triebwasser, Michael P; Ghiaccio, Valentina; Fedorky, Megan T; Gollomp, Kandace L; Bochenek, Veronica; Roche, Aoife M; Everett, John K; Cook, Emma J; Bushman, Frederic D; Teawtrakul, Nattiya; Glentis, Stavros; Kattamis, Antonis; Mui, Barbara L; Tam, Ying K; Weissman, Drew; Abdulmalik, Osheiza; Parhiz, Hamideh; Rivella, Stefano
细胞生物学
基因治疗
代谢
微生物学
干细胞
发育与干细胞
贫血
