TRANSCRIPTIONAL THERAPY BASED-LIPID NANOPARTICLES AND MRNA FOR THE TREATMENT OF END-STAGE LIVER DISEASE

Methods are disclosed for treating end-stage liver failure in a subject that include administering to the subject a therapeutically effective amount of a recombinant mRNA encoding HNF4α isoform 2. The methods can include administering a lipid nanoparticle including the recombinant mRNA to the subjec...

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Hauptverfasser:	SOTO-GUTIERREZ, Alejandro, FOX, Ira Jacob, ALAMEH, Mohamad-Gabriel, HAEP, Nils, DIAZ-ARAGON, Ricardo, WEISSMAN, Drew, FACCIOLI, Lanuza Alaby Pinheiro, FLORENTINO, Rodrigo Machado, OSTROWSKA, Alina Krystyna, TAFALENG, Edgar Naoe, BELL, Aaron W, MOTOMURA, Takashi
Format:	Patent
Sprache:	eng ; fre ; ger
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 ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Methods are disclosed for treating end-stage liver failure in a subject that include administering to the subject a therapeutically effective amount of a recombinant mRNA encoding HNF4α isoform 2. The methods can include administering a lipid nanoparticle including the recombinant mRNA to the subject. Also disclosed are recombinant mRNA encoding an HNF4α isoform 1 or HNF4α isoform 2, and lipid nanoparticles include the recombinant mRNA. Methods are also for treating liver disease or end-stage liver failure in a subject that include administering to the subject a therapeutically effective amount of a recombinant mRNA encoding HNF4α isoform 1.