USE OF NONSENSE CODON SUPPRESSOR COMPOUNDS FOR THE PREPARATION OF A MEDICAMENT FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY OR CYSTIC FIBROSIS

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专利权人:

PTC THERAPEUTICS; INC

发明人:

申请号：

IL222557

公开号：

IL222557A

申请日:

2012.10.18

申请国别(地区):

IL

年份:

2012

代理人:

摘要：

The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.

来源网站：

中国工程科技知识中心

来源网址：

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