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Updated: Jun 22, 2025

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
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在患有遗传性血管的患者中,C1抑制剂的正常化

Nicholas E Peters1, Dylan J Mac Lochlainn1, Fatima Dhalla1

  • 1From the Departments of Clinical Immunology (N.E.P., D.J.M.L., F.D., R.J., S.Y.P.) and Pediatric Gastroenterology (L.H.), Oxford University Hospitals NHS Foundation Trust, and the Institute of Developmental and Regenerative Medicine (F.D.) and the Department of Paediatrics (F.D., D.K.), University of Oxford, Oxford, and the Clinical Immunology Service, Institute of Immunology and Immunotherapy, College of Medical and Dental Sciences, University of Birmingham (N.E.P.), and the Liver Unit, Birmingham Women's and Children's Hospital (G.L.G., K.S.), Birmingham - all in the United Kingdom.

The New England journal of medicine
|July 3, 2024
PubMed
概括

遗传性血管,一种与SERPING1基因突变相关的疾病,在婴儿中通过肝移植可能被治愈. 该程序使C1抑制剂水平正常化,这表明了这种罕见的遗传疾病的新型治疗方法.

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