Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis - Qiu, Min; Tang, Yan; Chen, Jinjin; Muriph, Rachel; Ye, Zhongfeng; Huang, Changfeng; Evans, Jason; Henske, Elizabeth P.; Xu, Qiaobing (Proceedings of the National Academy of Sciences - PNAS, 2022)
Keywords: Animals; Biological Sciences; Drug Delivery Systems - methods; Drug development; Female; Gene Transfer Techniques; Genetic Engineering - methods; Lipids; Liposomes - chemistry; Liposomes - pharmacology; Liquid chromatography; Liver; Lung - cytology; Lung - pathology; Lung diseases; Lung Diseases - drug therapy; Lung Diseases - metabolism; Lymphangioleiomyomatosis - drug therapy; Lymphangioleiomyomatosis - metabolism; Mass spectrometry; Mass spectroscopy; Mice; Mice, Inbred BALB C; Mice, Inbred C57BL; mRNA; Mutation; Nanoparticles; Nanoparticles - chemistry; Organs; Physical Sciences; Plasma proteins; Protein Corona - chemistry; Protein Corona - metabolism; Proteins; Ribonucleic acid; RNA; RNA, Messenger - administration & dosage; RNA, Messenger - genetics; RNA, Messenger - pharmacology; RNA, Small Interfering - metabolism; Spleen; TSC2 gene; Tuberous sclerosis; Tuberous Sclerosis Complex 2; Tumor suppressor genes; Tumors; lipid nanoparticles; mRNA; tuberous sclerosis complex; lung-targeted delivery; lymphangioleiomyomatosis