[CRISPR作为血红蛋白病的功能性治疗]。

Q4 Medicine

Ugeskrift for laeger Pub Date : 2025-05-26 DOI:10.61409/V12240888

Andreas Glenthøj, Sarah Birgitte Ingemod Sand Carlsen, Marianne Hoffmann, Eva Kannik Haastrup, Lisbeth Pernille Andersen, Nina Toft, Brian Thomas Kornblit, Jesper Brix Petersen, Henrik Hasle, Marianne Rosenkrantz Segelcke Ifversen

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引用次数: 0

摘要

严重的血红蛋白病，包括镰状细胞病和β-地中海贫血，是严重的全球健康负担。CRISPR技术能够对造血干细胞进行精确的基因编辑，目前的治疗方法侧重于提高胎儿血红蛋白的产生，以实现功能性治愈。本综述发现，虽然体外方法很有前景，但需要先进的设施和大量的资源，限制了需求最高的地方的可及性。体内方法的未来发展可能会扩大全球可及性，解决对这些使人衰弱的疾病的可扩展和负担得起的治疗的迫切需求。

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[CRISPR as a functional cure for hemoglobinopathies].

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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来源期刊

Ugeskrift for laeger Medicine-Medicine (all)

CiteScore

0.20

自引率

0.00%

发文量

345

期刊介绍： The Ulster Medical Journal is an international general medical journal with contributions on all areas of medical and surgical specialties relevant to a general medical readership. It retains a focus on material relevant to the health of the Northern Ireland population. The Honorary Editor would welcome offers of papers for publication. Prospective authors are invited to read the notice to contributors.