CONCINO MICHAEL,HEARTLEIN MICHAEL,DEROSA FRANK,GUILD BRAYDON CHARLES
申请号：
NZ60061610
公开号：
NZ600616A
申请日:
2010.11.30
申请国别(地区):
NZ
年份:
2014
代理人:
摘要：
Disclosed is the use of a messenger RNA (mRNA) encoding an enzyme or a protein and encapsulated in a liposome in the manufacture of a medicament for treating a subject suffering from or susceptible to a deficiency of said enzyme or protein, wherein said medicament is formulated to provide prolonged stable expression of the enzyme or protein protein encoded by the mRNA in the liver, and wherein the liposome comprises one or more cationic lipids, one or more cholesterol based lipids and one or more PEG-modified lipids and has a size of less than 100 nm. Further disclosed is a composition formulated for in vivo production of protein comprising an mRNA encoding a protein, said mRNA encapsulated within a liposome, wherein the liposome comprises one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol based lipids and one or more PEG-modified lipids and has a size less than about 100 nm and wherein the composition is formulated to provide prolonged stable expression of the protein encoded by the mRNA in the liver.
