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Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
The New England Journal of Medicine ( IF 158.5 ) Pub Date : 2024-04-24 , DOI: 10.1056/nejmoa2309673 Franco Locatelli 1 , Peter Lang 1 , Donna Wall 1 , Roland Meisel 1 , Selim Corbacioglu 1 , Amanda M. Li 1 , Josu de la Fuente 1 , Ami J. Shah 1 , Ben Carpenter 1 , Janet L. Kwiatkowski 1 , Markus Mapara 1 , Robert I. Liem 1 , Maria Domenica Cappellini 1 , Mattia Algeri 1 , Antonis Kattamis 1 , Sujit Sheth 1 , Stephan Grupp 1 , Rupert Handgretinger 1 , Puja Kohli 1 , Daoyuan Shi 1 , Leorah Ross 1 , Yael Bobruff 1 , Christopher Simard 1 , Lanju Zhang 1 , Phuong Khanh Morrow 1 , William E. Hobbs 1 , Haydar Frangoul 1
The New England Journal of Medicine ( IF 158.5 ) Pub Date : 2024-04-24 , DOI: 10.1056/nejmoa2309673 Franco Locatelli 1 , Peter Lang 1 , Donna Wall 1 , Roland Meisel 1 , Selim Corbacioglu 1 , Amanda M. Li 1 , Josu de la Fuente 1 , Ami J. Shah 1 , Ben Carpenter 1 , Janet L. Kwiatkowski 1 , Markus Mapara 1 , Robert I. Liem 1 , Maria Domenica Cappellini 1 , Mattia Algeri 1 , Antonis Kattamis 1 , Sujit Sheth 1 , Stephan Grupp 1 , Rupert Handgretinger 1 , Puja Kohli 1 , Daoyuan Shi 1 , Leorah Ross 1 , Yael Bobruff 1 , Christopher Simard 1 , Lanju Zhang 1 , Phuong Khanh Morrow 1 , William E. Hobbs 1 , Haydar Frangoul 1
Affiliation
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)–Ca...
中文翻译：
Exagamglogene Autotemcel 治疗输血依赖性 β-地中海贫血
Exagamglogene autotemcel (exa-cel) 是一种非病毒细胞疗法，旨在通过离体成簇规则间隔短回文重复 (CRISPR)–Ca 重新激活胎儿血红蛋白合成。
更新日期：2024-04-25
中文翻译：
Exagamglogene Autotemcel 治疗输血依赖性 β-地中海贫血
Exagamglogene autotemcel (exa-cel) 是一种非病毒细胞疗法，旨在通过离体成簇规则间隔短回文重复 (CRISPR)–Ca 重新激活胎儿血红蛋白合成。
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