TRAITEMENT DE LA FIBROSE KYSTIQUE PAR ADMINISTRATION D'ARNM À CODONS OPTIMISÉS CODANT POUR LA CFTR

• 收藏
• 分享

专利权人:

Translate Bio; Inc.

发明人:

申请号：

EP18730550.3

公开号：

EP3624824A1

申请日:

2018.05.16

申请国别(地区):

EP

年份:

2020

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/