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Updated: Sep 18, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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[CRISPR as a functional cure for hemoglobinopathies]

Andreas Glenthøj1,2, Sarah Birgitte Ingemod Sand Carlsen1, Marianne Hoffmann3

  • 1Dansk Center for Røde Blodceller, Afdeling for Blodsygdomme, Københavns Universitetshospital - Rigshospitalet.

Ugeskrift for Laeger
|June 20, 2025
PubMed

Severe haemoglobinopathies, including sickle cell disease and β-thalassaemia, represent significant global health burdens. CRISPR technology enables precise genetic editing of haematopoietic stem cells, with current therapies focused on boosting fetal haemoglobin production for a functional cure. This review finds that, while promising, ex vivo approaches require advanced facilities and substantial resources, limiting accessibility where the need is highest. Future development of in vivo methods may expand global access, addressing the urgent need for scalable and affordable treatments for these debilitating diseases.

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