Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial
Blood（2022）
关键词
severe sickle cell disease,lentiviral gene therapy,fetal hemoglobin,shmir-based,post-transcriptional
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