CFTR MRNA COMPOSITIONS AND RELATED METHODS AND USES

Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis. La présente invention concerne des matériaux, des compositions...

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Hauptverfasser: GUILD, BRAYDON CHARLES, HEARTLEIN, MICHAEL, SMITH, LIANNE, PLANK, CHRISTIAN, DEROSA, FRANK, RUDOLPH, CARSTEN
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creator GUILD, BRAYDON CHARLES
HEARTLEIN, MICHAEL
SMITH, LIANNE
PLANK, CHRISTIAN
DEROSA, FRANK
RUDOLPH, CARSTEN
description Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis. La présente invention concerne des matériaux, des compositions, des procédés de production et des procédés d'administration d'ARNm du gène CFTR en vue de l'induction de l'expression de la protéine CFTR, y compris dans les poumons d'un mammifère. La présente invention se révèle particulièrement utile dans le cadre du traitement de la mucoviscidose.
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIREDCHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERSFROM A RACEMIC MIXTURE
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
title CFTR MRNA COMPOSITIONS AND RELATED METHODS AND USES
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