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US 11,389,547 B2Granted
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.
Claims19
A method comprising:
- replacing at least one uridine nucleotide of in vitro-synthesized RNA, comprising an open reading frame that encodes a recombinant protein of interest, with a nucleotide comprising the modified nucleoside 5-methoxyuridine (mo5U), thereby generating in vitro-synthesized RNA comprising mo5U in place of the at least one uridine nucleotide.
Dependencies are inferred from the claim text.