LIPIDIC NANOPARTICLES FOR MRNA DELIVERY

Disclosed herein are compositions for modulating the production of a protein in a target cell, in particular for ameliorating diseases associated with protein or enzyme deficiencies. The compositions comprise at least one mRNA encoding a polypeptide of interest, and a transfer vehicle comprising a l...

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Bibliographische Detailangaben
Hauptverfasser:	DEROSA, Frank, GUILD, Braydon Charles, HEARTLEIN, Michael
Format:	Patent
Sprache:	eng ; fre ; ger
Schlagworte:	HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
Online-Zugang:	Volltext bestellen
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Beschreibung
Zusammenfassung:	Disclosed herein are compositions for modulating the production of a protein in a target cell, in particular for ameliorating diseases associated with protein or enzyme deficiencies. The compositions comprise at least one mRNA encoding a polypeptide of interest, and a transfer vehicle comprising a lipid nanoparticle or a lipidoid nanoparticle.