Michael Heartlein,Frank DeRosa,Alan Kimura,Jonathan Abysalh,Anusha Dias,Shrirang Karve,Zarna Patel
申请号：
US15981757
公开号：
US20180333457A1
申请日:
2018.05.16
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.
