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| http://dx.doi.org/10.1016/j.gimo.2024.101875 | DOI Listing |
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Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel) for individuals 12 years and older with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOC): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).
Genet Med Open
September 2024
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Article Synopsis
- - Sickle cell disease (SCD) is a hereditary blood disorder that leads to chronic pain, anemia, and various health complications, but advancements in treatments have improved patient outcomes over time.
- - Despite progress, challenges like limited access to stem cell transplants and complications from current therapies highlight the need for ongoing research in SCD treatments.
- - The recent FDA approval of Casgevy, a CRISPR/Cas9-based gene therapy, represents a significant step in SCD treatment, with the review focusing on its potential effectiveness and safety compared to traditional methods, emphasizing the need for long-term research.
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Internal Medicine, Al Manhal Academy of Science, Sudan.
Sickle cell disease (SCD), the most common autosomal recessive genetic disorder, affects the hemoglobin (Hb) chains in human red blood cells. It is caused by mutations in the β-globin genes, leading to the production of hemoglobin S, which results in the formation of sickle-shaped red blood cells (RBCs). These abnormal cells cause hemolysis, endothelial damage, and small vessel occlusion, leading to both acute and long-term complications.
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