TY - JOUR
T1 - CRISPR som funktionel kur af hæmoglobinsygdomme
AU - Glenthøj, Andreas
AU - Carlsen, Sarah Birgitte Ingemod Sand
AU - Hoffmann, Marianne
AU - Haastrup, Eva Kannik
AU - Andersen, Lisbeth Pernille
AU - Toft, Nina
AU - Kornblit, Brian Thomas
AU - Petersen, Jesper Brix
AU - Hasle, Henrik
AU - Ifversen, Marianne Rosenkrantz Segelcke
N1 - Published under Open Access CC-BY-NC-BD 4.0. https://creativecommons.org/licenses/by-nc-nd/4.0/.
PY - 2025/5/26
Y1 - 2025/5/26
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=105009646275&partnerID=8YFLogxK
U2 - 10.61409/V12240888
DO - 10.61409/V12240888
M3 - Review
C2 - 40539281
SN - 0041-5782
VL - 187
JO - Ugeskrift for Laeger
JF - Ugeskrift for Laeger
IS - 22
M1 - V12240888
ER -