Exagamglogene Autotemcel for Severe Sickle Cell Disease

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Exagamglogene Autotemcel for Severe Sickle Cell Disease

Frangoul, Haydar; Locatelli, Franco; Sharma, Akshay; Bhatia, Monica; Mapara, Markus; Molinari, Lyndsay; Wall, Donna; Liem, Robert I.; Telfer, Paul; Shah, Ami J.; Cavazzana, Marina; Corbacioglu, Selim; Rondelli, Damiano; Meisel, Roland; Dedeken, Laurence; Lobitz, Stephan; de Montalembert, Mariane; Steinberg, Martin H.; Walters, Mark C.; Eckrich, Michael J.; Imren, Suzan; Bower, Laura; Simard, Christopher; Zhou, Weiyu; Xuan, Fengjuan; Morrow, Phuong Khanh; Hobbs, William E.; Grupp, Stephan A.

The New England journal of medicine, 05/2024, Volume: 390, Issue: 18

Journal Article

Of 30 patients with severe sickle cell disease who were treated with gene-edited autologous hematopoietic stem and progenitor cells, 29 were free from vaso-occlusive crises for at least 12 consecutive months.

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