发明名称 |
MRNA THERAPY FOR UREA CYCLE DISORDERS |
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 |
US2014294940(A1) |
申请公布日期 |
2014.10.02 |
申请号 |
US201414309387 |
申请日期 |
2014.06.19 |
申请人 |
Shire Human Genetic Therapies, Inc. |
发明人 |
Guild Braydon Charles;DeRosa Frank;Heartlein Michael |
分类号 |
A61K48/00 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
Lexington MA US |