发明名称 |
PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM |
摘要 |
The present invention relates to gene therapy systems designed for the delivery of a therapeutic product to a subject using replication-defective virus composition(s) engineered with a built-in safety mechanism for ablating the therapeutic gene product, either permanently or temporarily, in response to a pharmacological agent - preferably an oral formulation, e.g., a pill. The invention is based, in part, on the applicants' development of an integrated approach, referred to herein as "PITA" (Pharmacologically Induced Transgene Ablation), for ablating a transgene or negatively regulating transgene expression. In this approach, replication-deficient viruses are used to deliver a transgene encoding a therapeutic product (an RNA or a protein) so that it is expressed in the subject, but can be reversibly or irreversibly turned off by administering the pharmacological agent; e.g., by administration of a small molecule that induces expression of an ablator specific for the transgene or its RNA transcript. |
申请公布号 |
WO2011126808(A2) |
申请公布日期 |
2011.10.13 |
申请号 |
WO2011US30213 |
申请日期 |
2011.03.28 |
申请人 |
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA;WILSON, JAMES, M.;CHEN, SHU-JEN;TRETIAKOVA, ANNA, P. |
发明人 |
WILSON, JAMES, M.;CHEN, SHU-JEN;TRETIAKOVA, ANNA, P. |
分类号 |
C12N15/86;A61K48/00;C12N15/864 |
主分类号 |
C12N15/86 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|