Regulatory Assessment of Casgevy for the Treatment of Transfusion-Dependent β-Thalassemia and Sickle Cell Disease with Recurrent Vaso-Occlusive Crises

Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are hereditary haemoglobinopathies characterized by a reduction in functional β-globin chains. Both conditions cause tiredness and increase susceptibility to infection, which can lead organ failure, significantly reducing life e...

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Egile Nagusiak:	Essam Kerwash, Marija Sajic, Khadija Rerhou Rantell, James W. McBlane, John D. Johnston, Alison Niewiarowska, Andrew S. Butler, Susan Cole
Formatua:	Artigo
Hizkuntza:	Inglês
Argitaratua:	MDPI AG 2024-07-01
Saila:	Current Issues in Molecular Biology
Gaiak:	sickle cell disease transfusion-dependent β-thalassemia 2 Casgevy CRIPSR/Cas9 technology
Sarrera elektronikoa:	https://www.mdpi.com/1467-3045/46/8/485
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