基于细胞的 b-地中海贫血症基因治疗。

Cell-Based Gene Therapy for b-Thalassemia.

机构信息

Department of Pediatrics, Maulana Azad Medical College (University of Delhi) and associated Lok Nayak Hospital, New Delhi. Correspondence to: Dr Yajika Arya, Senior Resident, Department of Pediatrics, Lok Nayak Hospital, New Delhi 110 002.

Department of Pediatrics, Maulana Azad Medical College (University of Delhi) and associated Lok Nayak Hospital, New Delhi.

出版信息

Indian Pediatr. 2023 Apr 15;60(4):313-316.

PMID:37002845

Abstract

The United States Food and Drug Administration (FDA) approved betibeglogene autotemcel (beti-cel), the first cell-based gene therapy for adult and pediatric patients with b-thalassemia in August, 2022. This update details this and other novel therapies that have emerged in the treatment of b-thalassemia, apart from transfusion and iron chelation, with particular focus on newly approved gene therapy.

摘要

美国食品和药物管理局（FDA）于 2022 年 8 月批准了 betibeglogene autotemcel（beti-cel），这是第一种用于治疗成人和儿科 b-地中海贫血患者的基于细胞的基因疗法。本更新详细介绍了除输血和铁螯合之外，在 b-地中海贫血治疗中出现的其他新型疗法，特别关注新批准的基因疗法。