Inventors:
Drew Weissman - Wynnewood PA, US
Assignee:
The Trustees Of the University of Pennsylvania - Philadelphia PA
International Classification:
C07H 21/02
US Classification:
514 44 R, 435 691, 435 61, 435 694, 435189, 435207, 435 6952, 435192, 435 792, 435 611
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.