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Delivery Of mRNA For The Augmentation Of Proteins And Enzymes In Human Genetic Diseases
专利权人:
Shire Human Genetic Therapies
发明人:
Guild, Braydon Charles,DeRosa, Frank,Heartlein, Michael,Concino, Michael
申请号:
AU2014250713
公开号:
AU2014250713B2
申请日:
2014.10.17
申请国别(地区):
AU
年份:
2016
代理人:
摘要:
#$%^&*AU2014250713B220160728.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
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