Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches

Sickle cell disease (SCD) is an inherited blood disorder marked by the production of abnormal hemoglobin, leading to the distortion—or sickling—of red blood cells. The SCD arises from a single-point mutation that substitutes glutamic acid with valine at the sixth codon of the β-globin chain in hemog...

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Main Authors:	Bakhtiar, M. Taher, Aminondin, Sofea 'Aisyah, Nasir, Nur Asyilah, Jasmadi, Noor Afiqah, Nizam, Nur Irdeena Nabella, Shahrul, Ilhan Syahmi, Darnis, Deny Susanti, Khotib, Junaidi, Faiyazuddin, Md, Widodo, Riyanto Teguh, Haris, Muhammad Salahuddin
Format:	Article
Language:	en en en
Published:	Frontiers Media S.A. 2025
Subjects:	RS Pharmacy and materia medica
Online Access:	http://irep.iium.edu.my/122896/1/122896_Sickle%20cell%20disease.pdf http://irep.iium.edu.my/122896/7/122896_Sickle%20cell%20disease_SCOPUS.pdf http://irep.iium.edu.my/122896/8/122896_Sickle%20cell%20disease_WOS.pdf http://irep.iium.edu.my/122896/ https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1630994/full
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