| Autor: |
Per H. Basse, Anand K. Katakam, Steve Goding, Prasanna Kumar, Qin Yang |
| Rok vydání: |
2006 |
| Předmět: |
|
| Zdroj: |
Molecular Therapy. 13 |
| ISSN: |
1525-0016 |
| DOI: |
10.1016/j.ymthe.2006.08.324 |
| Popis: |
Some gene therapy approaches will require the therapeutic gene to be expressed in a regulated fashion so that the precise amount of the gene product can be delivered in the appropriate tissues at the desired time and for the desired duration. We have developed the RheoSwitch|[reg]| Therapeutic System (RTS) that can be precisely controlled by an orally active synthetic small molecule ligand (Activator Drug) for regulated expression of therapeutic genes. The RTS consists of two component proteins that heterodimerize to create a functional inducible transcription factor complex. The ligand- binding component is the DEF domain of a mutant ecdysone receptor that is fused to a Gal4 DNA binding domain. The second component is the EF domain of a chimeric RXR that is fused to the VP16 activation domain. These proteins, constitutively expressed under a ubiquitous or tissue-specific promoter, can induce in vivo gene expression from a responsive promoter in the presence of a potent ligand. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
|
