Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference
通过 HBBAS3 基因添加和突变特异性 RNA 干扰对 β-地中海贫血进行治疗的单体和双功能 GLOBE 载体的评估
期刊:Cells
影响因子:5.1
doi:10.3390/cells12242848
Lola Koniali, Christina Flouri, Markela I Kostopoulou, Nikoletta Y Papaioannou, Panayiota L Papasavva, Basma Naiisseh, Coralea Stephanou, Anthi Demetriadou, Maria Sitarou, Soteroula Christou, Michael N Antoniou, Marina Kleanthous, Petros Patsali, Carsten W Lederer