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:US20160199357A1
公开(公告)日:2016-07-14
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意义突变的核酸序列编码的功能蛋白质。本发明还涉及用于生产由包含无意义突变的核酸序列编码的功能蛋白质的方法,并使用这些蛋白质预防、管理和/或治疗与基因中的无意义突变相关的疾病。