The invention relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR), isoforms thereof, functional fragments thereof, and fusion proteins comprising CFTR. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of CFTR expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient CFTR activity in subjects.
该发明涉及mRNA治疗囊性纤维化的方法。该发明中使用的mRNAs在体内施用时,编码囊性纤维化跨膜传导调节蛋白(CFTR)、其同工型、功能性片段以及包含CFTR的融合蛋白。该发明中的mRNAs最好被封装在脂质纳米粒子(LNPs)中,以实现有效地传递到受试者的细胞和/或组织,当施用给受试者时。该发明的mRNA治疗方法增加和/或恢复受试者中CFTR表达和/或活性的不足
水平。该发明的mRNA治疗方法进一步降低与受试者中CFTR活性不足相关的有毒代谢物的
水平。