Modified lentiviral globin gene therapy for pediatric β0/β0 transfusion-dependent β-thalassemia: A single-center, single-arm pilot trial

Author:

Li Shiqi,Ling Sikai,Wang Dawei,Wang Xiaoyuan,Hao Fangyuan,Yin Liufan,Yuan Zhongtao,Liu Lin,Zhang Lin,Li Yu,Chen Yingnian,Luo Le,Dai Ying,Zhang Lihua,Chen Lvzhe,Deng Dongjie,Tang Wei,Zhang Sujiang,Wang Sanbin,Cai YujiaORCID

Publisher

Elsevier BV

Reference42 articles.

1. β-Thalassemia;Origa;Genet. Med.,2017

2. The Present and Future Global Burden of the Inherited Disorders of Hemoglobin;Piel;Hematol. Oncol. Clin. North Am.,2016

3. Thalassaemia;Taher;Lancet,2018

4. The Molecular Basis of β-Thalassemia;Thein;Cold Spring Harbor Perspect. Med.,2013

5. Long-term results of survival in patients with thalassemia major treated with bone marrow transplantation;Di Bartolomeo;Am. J. Hematol.,2008

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