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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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Principais autores: Muhammad Taher, Sofea ‘Aisyah Aminondin, Nur Asyilah Nasir, Noor Afiqah Jasmadi, Nur Irdeena Nabella Nizam, Ilhan Syahmi Shahrul, Deny Susanti, Junaidi Khotib, Md Faiyazuddin, Riyanto Teguh Widodo, Muhammad Salahuddin Haris
Formato: Artigo
Idioma:Inglês
Publicado em: Frontiers Media S.A. 2025-08-01
Colecção:Frontiers in Pharmacology
Assuntos:
anemia
gene therapy
gene editing
CRISPR
hemoglobinopathies
hematopoietic stem cell transplantation
Acesso em linha:https://www.frontiersin.org/articles/10.3389/fphar.2025.1630994/full
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