The present invention relates to an RNA encoding a therapeutic protein. In particular, the present invention relates to RNA suitable for use as a medicament. The present invention concerns such novel RNA as well as compositions and kits comprising the RNA. Furthermore, the present invention relates to the RNA, compositions or kits as disclosed herein for use as a medicament. The present invention also provides the use of the RNA, compositions or kits as disclosed herein far increasing the expression of said encoded protein, in particular in gene therapy.
本发明涉及一种编码治疗蛋白质的 RNA。特别是,本发明涉及适合用作药物的 RNA。本发明涉及这种新型 RNA 以及包含该 RNA 的组合物和试剂盒。此外,本发明还涉及本文公开的用作药物的 RNA、组合物或试剂盒。本发明还提供了本文所公开的 RNA、组合物或试剂盒在提高所述编码蛋白表达量方面的用途,特别是在
基因治疗中的用途。