CHEMICALLY MODIFIED MESSENGER RNA'S

This invention provides messenger RNA (mRNA) molecules comprising comprising an open reading frame that encodes a protein of interest, wherein said modified RNA comprises a modified nucleoside selected from the group consisting of: (I), (II), and (III), gene therapy vectors comprising same, methods...

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Hauptverfasser: NAKAJIMA, Katsumasa, CAPLAN, Shari Lynn
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creator NAKAJIMA, Katsumasa
CAPLAN, Shari Lynn
description This invention provides messenger RNA (mRNA) molecules comprising comprising an open reading frame that encodes a protein of interest, wherein said modified RNA comprises a modified nucleoside selected from the group consisting of: (I), (II), and (III), 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 mRNA molecules. La présente invention concerne des molécules d'ARN messager (ARNm) comprenant un cadre de lecture ouvert qui code pour une protéine d'intérêt, ledit ARN modifié comprenant un nucléoside sélectionné dans le groupe constitué de : (I), (II), et (III), des vecteurs de thérapie génique les comprenant, des procédés de synthèse associés, et des procédés de remplacement génique, de thérapie génique, de silençage de la transcription génique et d'administration de protéines thérapeutiques au tissu in vivo, comprenant les molécules. La présente invention concerne également des procédés de réduction de l'immunogénicité de molécules d'ARNm.
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The present invention also provides methods of reducing the immunogenicity of mRNA molecules. La présente invention concerne des molécules d'ARN messager (ARNm) comprenant un cadre de lecture ouvert qui code pour une protéine d'intérêt, ledit ARN modifié comprenant un nucléoside sélectionné dans le groupe constitué de : (I), (II), et (III), des vecteurs de thérapie génique les comprenant, des procédés de synthèse associés, et des procédés de remplacement génique, de thérapie génique, de silençage de la transcription génique et d'administration de protéines thérapeutiques au tissu in vivo, comprenant les molécules. 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The present invention also provides methods of reducing the immunogenicity of mRNA molecules. La présente invention concerne des molécules d'ARN messager (ARNm) comprenant un cadre de lecture ouvert qui code pour une protéine d'intérêt, ledit ARN modifié comprenant un nucléoside sélectionné dans le groupe constitué de : (I), (II), et (III), des vecteurs de thérapie génique les comprenant, des procédés de synthèse associés, et des procédés de remplacement génique, de thérapie génique, de silençage de la transcription génique et d'administration de protéines thérapeutiques au tissu in vivo, comprenant les molécules. 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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
DERIVATIVES THEREOF
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
SUGARS
VINEGAR
WINE
title CHEMICALLY MODIFIED MESSENGER RNA'S
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