Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding CFTR

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专利权人:

Translate Bio; Inc.

发明人:

Heartlein, Michael,Derosa, Frank,Kimura, Alan,Abysalh, Jonathan,Dias, Anusha,Karve, Shrirang,Patel, Zarna

申请号：

AU2018268859

公开号：

AU2018268859A1

申请日:

2018.05.16

申请国别(地区):

AU

年份:

2019

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.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/