Gene editing flows to the lungs
成果类型：
Editorial Material
署名作者：
Bulcaen, Mattijs; Carlon, Marianne S.
署名单位：
KU Leuven
刊物名称：
SCIENCE
ISSN/ISSBN：
0036-11628
DOI：
10.1126/science.adq0059
发表日期：
2024-06-14
页码：
1175-1176
关键词：
摘要：
The lungs encompass a vast surface for gas exchange-they are the bellows that keep the body oxygenated and its metabolism burning. When problems arise in the lungs, severe diseases follow, such as cystic fibrosis. Development of gene therapies for cystic fibrosis has been challenging, especially because delivery usually involves inhalation. Permanently exposed to the hostile outside environment, the lungs are equipped with defense mechanisms to keep inhaled particles and pathogens out of the lung cells. More than 27 clinical trials for cystic fibrosis gene therapy have failed to find a way past the airway epithelial barrier ( 1 ). On page 1196 of this issue, Sun et al. ( 2 ) report intravenous airway targeting using lung-targeted lipid nanoparticles (LNPs) containing CRISPR gene editing tools in a mouse model of cystic fibrosis. This basolateral route might provide a breach through the airway barriers to treat cystic fibrosis and possibly other lung diseases.