Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of Β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference.
CELLS（2023）
Key words
gene therapy,RNA interference,shRNAmiR,lentiviral vector,hemoglobinopathy,thalassemia,sickle cell anemia
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