Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith
申请人:PTC Therapeutics, Inc.
公开号:EP2387995A1
公开(公告)日:2011-11-23
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.
本发明涉及由包含无义突变的核酸序列编码的功能性蛋白质。本发明还涉及生产由包含无义突变的核酸序列编码的功能性蛋白质的方法,以及将这种蛋白质用于预防、控制和/或治疗与基因中的无义突变有关的疾病的方法。