COMPOSES DE TRINUCLEOTIDE PRESENTANT UNE COIFFE EN POSITION 5' OU COMPOSES D'OLIGONUCLEOTIDE DE RANG SUPERIEUR ET LEURS UTILISATIONS DANS LA STABILISATION D'ARN, L'EXPRESSION DE PROTEINE ET EN THERAPIE
KUHN, ANDREAS,MURAMATSU, HIROMI,KARIKO, KATALIN,FESSER, STEPHANIE,SAHIN, UGUR
申请号：
CA3093509
公开号：
CA3093509A1
申请日:
2019.03.14
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
The present invention relates to 5'-cap compounds, in particular the stabilization of RNA by such 5'-cap compounds, and provides compositions, such as pharmaceutical compositions, and cells comprising an RNA which is modified with such a 5 '-cap compound, as well as methods for producing a peptide or protein of interest using the compositions or cells according to the present invention. Furthermore, the present invention provides the RNA, compositions, or cells for use in therapy, in particular for use in a method of treating a disease or disorder by protein replacement therapy, genome engineering, genetic reprogramming, and immunotherapy； a method for increasing the stability of RNA in cells； a method for increasing the expression of RNA in cells； and a method for providing an RNA with a 5'-cap structure.
