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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主要な著者:	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
フォーマット:	Artigo
言語:	Inglês
出版事項:	Frontiers Media S.A. 2025-08-01
シリーズ:	Frontiers in Pharmacology
主題:	anemia gene therapy gene editing CRISPR hemoglobinopathies hematopoietic stem cell transplantation
オンライン･アクセス:	https://www.frontiersin.org/articles/10.3389/fphar.2025.1630994/full
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