A breath of fresh air:targeted non-viral in vivo gene correction in the mammalian lung  

作　　者：Jixin Liu Dirk Grimm 

机构地区：[1]Department of Infectious Diseases/Virology,Section Viral Vector Technologies,Medical Faculty,University of Heidelberg,BioQuant,Center for Integrative Infectious Diseases(CIID),69120 Heidelberg,Germany [2]German Center for Infection Research(DZIF)and German Center for Cardiovascular Research(DZHK),partner site Heidelberg,69120 Heidelberg,Germany [3]Faculty of Engineering Sciences,University of Heidelberg,69120 Heidelberg,Germany

出　　处：《Signal Transduction and Targeted Therapy》2024年第11期4771-4772,共2页信号转导与靶向治疗（英文）

基　　金：D.G.appreciates support by the German Research Foundation(DFG)through the DFG Collaborative Research Centers SFB1129(Projektnummer 240245660);TRR179(Projektnummer 272983813).

摘　　要：In a recent study published in Science,1 Sun and colleagues showcase the power and potential of lung SORT LNPs,i.e.,lipid nanoparticles that upon systemic delivery in mice specifically and efficiently target cells in the lung,most likely facilitated by their binding to plasma vitronectin and uptake via the vitronectin receptor.Most remarkably,when engineered to deliver a base editor,peripheral injection of SORT LNPs enabled highly efficient gene correction in lung stem cells,whole lung and trachea in a mouse model of cystic fibrosis,illustrating the enormous promise of this novel technology for human patients suffering from this devastating disease(Fig.1).

关 键 词：LUNG CORRECTION EDITOR 

分 类 号：R394[医药卫生—医学遗传学]