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

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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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-08-01
Series:Frontiers in Pharmacology
Subjects:
anemia
gene therapy
gene editing
CRISPR
hemoglobinopathies
hematopoietic stem cell transplantation
Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2025.1630994/full
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https://www.frontiersin.org/articles/10.3389/fphar.2025.1630994/full

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