This invention provides messenger RNA (mRNA) molecules comprising an open reading frame that encodes a protein of interest, wherein said modified RNA comprises a modified nucleoside selected from the group consisting of: (I), (II), and (III), gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of mRNA molecules.
本发明提供了信使 RNA(mRNA)分子,该分子包含编码感兴趣的蛋白质的开放阅读框,其中所述修饰的 RNA 包含选自由(Ⅰ)、(Ⅱ)和(Ⅲ)组成的组的修饰核苷、包含该分子的
基因治疗载体、合成该分子的方法,以及包含该分子的
基因替换、
基因治疗、
基因转录沉默和向体内组织递送治疗蛋白质的方法。本发明还提供了降低 mRNA 分子免疫原性的方法。