Delivery of mRNA for the augmentation of proteins and enzymes in human genetic diseases
摘要
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.
申请公布号
AU2010326132(B2)
申请公布日期
2014.08.21
申请号
AU20100326132
申请日期
2010.11.30
申请人
SHIRE HUMAN GENETIC THERAPIES
发明人
GUILD, BRAYDON CHARLES;DEROSA, FRANK;HEARTLEIN, MICHAEL;CONCINO, MICHAEL