发明名称 RIBONUCLEIC ACIDS WITH 4'-THIO-MODIFIED NUCLEOTIDES AND RELATED METHODS
摘要 Disclosed are messenger RNA molecules and related compositions incorporating a 4′-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders. In certain embodiments, the 4′-thio modified mRNA provides for enhanced stability and/or reduced immunogenicity in in vivo therapies.
申请公布号 EP2970351(A1) 申请公布日期 2016.01.20
申请号 EP20140721635 申请日期 2014.03.14
申请人 SHIRE HUMAN GENETIC THERAPIES, INC. 发明人 DEROSA, FRANK;HEARTLEIN, MICHAEL
分类号 C12N15/67 主分类号 C12N15/67
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