Disclosed herein are compositions for use in am method of treating a disease in a subject, wherein the disease is due to a deficiency of a pulmonary surfactant-associated protein. The composition comprises mRNAs encoding the pulmonary surfactant-associated protein and a lipid nanoparticle that comprises cationic lipids, anionic lipids and PEG-modified lipids. The composition is to be administered via the pulmonary route.
本文公开了用于治疗受试者疾病的方法的组合物,其中疾病是由于肺表面活性物质相关蛋白的缺乏引起的。组合物包括编码肺表面活性物质相关蛋白的 mRNA 和脂质纳米颗粒,脂质纳米颗粒包括阳离子脂质、阴离子脂质和 P
EG 改性脂质。该组合物通过肺部途径给药。