Guild, Braydon Charles,DeRosa, Frank,Heartlein, Michael,Concino, Michael
申请号：
AU2016250459
公开号：
AU2016250459B2
申请日:
2016.10.28
申请国别(地区):
AU
年份:
2018
代理人:
摘要：
#$%^&*AU2016250459B220180628.pdf#####ABSTRACT (FIG. 1) Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.1/6 OH N DCC/DMAP N / -- P hHO Ph Ph 1 2 0 0 TFA/Anisole/DCM AO ,0 ' N IN>； 4 31 N NH Imidazole / --PhCholesterol Ph Ester (ICE) FIG. 1 Mean Firefly luciferase activity in livers of WT mice 4 hr post IV injection mRNA.31.10 4000030000- : 5926-12 .20000j 6031-190 10000 [M 6031-21Q 600o0 Untreated 40002000300Liver Spleen Tissue Sample FIG. 2
