Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cells

被引：0

作者：

Isabelle Rose ^{[1
]}

Miriam Greenwood ^{[2
]}

Matthew Biggart ^{[3
]}

Natalie Baumlin ^{[4
]}

Robert Tarran ^{[3
]}

Stephen L. Hart ^{[2
]}

Deborah L. Baines ^{[1
]}

机构：

[1] City St George’s,Institute of Infection and Immunity, School of Health and Medical Sciences

[2] University of London,Department of Genetics and Genomic Medicine

[3] UCL Great Ormond Street Institute of Child Health,Division of Genetic, Environmental and Inhalational Disease

[4] University of Kansas Medical Center,Department of Internal Medicine

[5] University of Kansas Medical Center,undefined

来源：

关键词：

Class 1 mutation; Transfection; Air–liquid-interface; Nasal; Ciliated cell; Secretory cell;

D O I：

10.1007/s00018-025-05587-y

中图分类号：

学科分类号：

摘要：

引用

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