发明名称 |
TFEB GENE THERAPY OF ALPHA-1-ANTITRYPSIN DEFICIENCY |
摘要 |
The present invention refers to a vector for gene therapy comprising a TFEB coding sequence under the control of a promoter able to efficiently express said TFEB coding sequence, to host cell comprising said vector and to their use in the gene therapy of a pathological condition characterized by a deficiency of alpha-1-antitrypsin (AAT). The present invention also refers to a pharmaceutical composition comprising the vector or the host cell of the invention for gene therapy and to a method for gene therapy of a pathological condition characterized by a deficiency of alpha-1-antitrypsin (AAT). |
申请公布号 |
EP2838567(A1) |
申请公布日期 |
2015.02.25 |
申请号 |
EP20130717291 |
申请日期 |
2013.04.19 |
申请人 |
FONDAZIONE TELETHON |
发明人 |
BRUNETTI-PIERRI, NICOLA;BALLABIO, ANDREA;PASTORE, NUNZIA |
分类号 |
A61K48/00;A61P1/16;C12N15/861 |
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 |
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