POLYNUCLEOTIDES ENCODING CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS

This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) when administered in vivo encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs...

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Bibliographische Detailangaben
Hauptverfasser:	TATE, Daniel, CRAWFORD, Matthew, MCLAUGHLIN, Christopher Karl, SUNG, Jean C, METKAR, Mihir, SEEPERSAUD, Mohindra, MIHAI, Cosmin, CHENEY, Jessica
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) when administered in vivo encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects. La présente divulgation concerne des véhicules d'administration comprenant des molécules de charge utile, par exemple, un ARNm pour le traitement de la mucoviscidose (MV). Des agents thérapeutiques à base d'acides nucléiques (par exemple, des ARNm) lorsqu'ils sont administrés in vivo codent pour le régulateur de la conductance transmembranaire de la mucoviscidose (CFTR). Les agents thérapeutiques à base d'acide nucléiques (par ex. les ARNm) de la divulgation augmentent et/ou restaurent des niveaux déficients d'expression et/ou d'activité de la CFTR chez les sujets.