Crispr/Cas9 gene editing in transfusion-dependent β-thalassemia and sickle cell disease: a systematic review of non-randomized clinical trials

Abstract Background β-Thalassemia and sickle cell disease are the most common hereditary β-hemoglobinopathies. The current available treatments for their severe cases include frequent blood transfusions, iron chelation therapy, and allogenic hematopoietic stem cell transplantation (HSCT). Clustered...

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Bibliografski detalji

Glavni autori:	Basma M. El-Khalifa, Weaam A. Soliman, Hazem M. Mazy, Muhtadi G. Ahmed, Hamza Al-Trad, Tarek H. Elmeneizee, Hossam W. Abd El-Aziz, Selma Ahmed Hassan Abdelmutaleb, Ghada A. Abdelghaffar, Maha H. Morsi
Format:	Artigo
Jezik:	Inglês
Izdano:	SpringerOpen 2025-11-01
Serija:	Egyptian Journal of Medical Human Genetics
Teme:	β-Thalassemia Crispr/Cas9 Gene editing Sickle cell disease
Online pristup:	https://doi.org/10.1186/s43042-025-00806-4
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