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<p>Sickle Cell Disease (SCD) remains a significant global health burden, disproportionately affecting low- and middle-income countries (LMICs), particularly in sub-Saharan Africa. Recent advancements in gene-based therapies, including CRISPR-Cas9 technologies like Casgevy and lentiviral treatments like Lyfgenia, offer promising curative solutions. However, these therapies are challenged by high costs, limited accessibility, and safety concerns, such as off-target effects and immune responses. This review emphasizes the need for sustainable financing models, such as global funding frameworks and public–private partnerships, to make these therapies affordable and accessible. Strengthening healthcare infrastructure, fostering global research collaborations, and community involvement are critical strategies to bridge existing gaps in SCD care. Additionally, ethical considerations and regulatory frameworks must evolve to ensure safe and equitable use of gene-editing technologies. A comprehensive, multi-sectoral approach is essential to transform SCD management, reduce mortality, and improve the quality of life for affected populations worldwide.</p>

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Gene based therapy for sickle cell disease in low and middle income countries insights from the Gambia

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年5月2日

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  • Rangarirai Makuku,
  • Fiona McGowan Martha Hutton,
  • Leander Penaso Marquez,
  • Sunny O. Abarikwu,
  • Marcarious M. Tantuoyir,
  • Muhammed Camara,
  • Momodou S. Jallow,
  • Ernest Moyo,
  • Lindelwa Phakamile Mmema,
  • Michael P. K. Mendy,
  • Kelvin Thomas,
  • Emmanuel Lawal

摘要

Sickle Cell Disease (SCD) remains a significant global health burden, disproportionately affecting low- and middle-income countries (LMICs), particularly in sub-Saharan Africa. Recent advancements in gene-based therapies, including CRISPR-Cas9 technologies like Casgevy and lentiviral treatments like Lyfgenia, offer promising curative solutions. However, these therapies are challenged by high costs, limited accessibility, and safety concerns, such as off-target effects and immune responses. This review emphasizes the need for sustainable financing models, such as global funding frameworks and public–private partnerships, to make these therapies affordable and accessible. Strengthening healthcare infrastructure, fostering global research collaborations, and community involvement are critical strategies to bridge existing gaps in SCD care. Additionally, ethical considerations and regulatory frameworks must evolve to ensure safe and equitable use of gene-editing technologies. A comprehensive, multi-sectoral approach is essential to transform SCD management, reduce mortality, and improve the quality of life for affected populations worldwide.