RNA containing modified nucleosides and methods of use thereof
AssigneeThe Trustees of the University of Pennsylvania
InventorsKatalin Kariko, Drew Weissman
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Abstract
This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, 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 RNA, oligoribonucleotide, and polyribonucleotide molecules.
Claims21
A composition comprising an in vitro-synthesized modified RNA comprising an open reading frame that encodes a protein of interest for translation in a mammalian cell, wherein said in vitro-synthesized modified RNA comprises a modified nucleoside selected from the group consisting of (i) 1-methypseudouridine (m1Ψ) and (ii) pseudouridine (Ψ).
Dependencies are inferred from the claim text.