abstract |
IT REFERS TO A COMPOSITION THAT INCLUDES AN mRNA THAT CODES A PROTEIN SUCH AS ARGININOSUCCINATE SYNTHETASE (ASS1), FACTOR IX, PHENYLALANINE HYDROXYLASE, AMONG OTHERS, WHICH IS ENCAPSULATED WITH A LIPEOMED FOR LIPEOMED IN A LIPE-CAPSULATE CAPSULATE i) p, R2, R6 AND R7 ARE AS DEFINED IN THE DESCRIPTION, THE CKK-E12 LIPID BEING PREFERRED; ii) NON-CATIONIC LIPIDS SUCH AS 1,2-DIOLEYL-SN-GLYCERO-3-PHOSPHOETHANOLAMINE (DOPE), 1,2-DIPALMITOYL-SN-GLYCERO-3-PHOSPHOCOLINE (DPPC), AMONG OTHERS; iii) CHOLESTEROL-BASED LIPIDS SUCH AS CHOLESTEROL OR PEGILATED CHOLESTEROL; AND iv) PEG-MODIFIED LIPIDS SUCH AS DMG-PEG2K; BEING A PREFERRED COMBINATION OF LIPIDS: CKK-E12, DOPE, CHOLESTEROL AND DMG-PEG2K. IT ALSO REFERS TO A METHOD OF ADMINISTRATION OF SUCH COMPOSITION |