In:
Science Translational Medicine, American Association for the Advancement of Science (AAAS), Vol. 14, No. 673 ( 2022-11-30)
Kurzfassung:
Cutting down blood clots with CrisprDespite lifelong anticoagulant treatment, thrombotic events are still common in patients with antithrombin (AT) deficiency. Here Tang and colleagues used Crispr-Cas9 gene editing to correct a 〈 italic 〉 SERPINC1 〈 /italic 〉 mutation in induced pluripotent stem cell (iPSC) hepatocytes derived from AT patient macrophages. Injection of these hepatocytes into spleens of AT knockout mice normalized plasma AT concentration and reduced thrombus formation for up to three weeks. These findings indicate that delivery of gene-edited iPSCs may be a promising strategy to improve coagulation in AT deficient patients. -- AW
Materialart:
Online-Ressource
ISSN:
1946-6234
,
1946-6242
DOI:
10.1126/scitranslmed.abq3202
Sprache:
Englisch
Verlag:
American Association for the Advancement of Science (AAAS)
Publikationsdatum:
2022
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