Understanding Biological Activity/Target Validation

To better understand how your gene of interest functions in vivo, you can use ReGenX AAV Vector Technology to overexpress your gene of interest in an appropriate tissue, to express a microRNA, or to knock down gene expression using short hairpin (shRNA, a form of RNAi). This can be accomplished in normal animals, or in disease/mutant models, such as transgenic or knockout mice, without the time required for cross-breeding animals. The creation of a ReGenX AAV vector is faster than the creation of a new transgenic or knockout mouse line, and eliminates issues relating to transgene expression (knockdown) during fetal development. One ReGenX AAV vector can be used in multiple animal models, and in multiple species, including mouse, rat, hamster, rabbit, cat, dog, and non-human primates.

With ReGenX AAV Vector Technology, you can selectively target different tissues, depending on the serotype selected, route of administration, and choice of promoter. Liver can be used as a depot for systemic expression of secreted proteins. High levels of transgene expression can be achieved in most tissues.

In summary, you can:

• Analyze the biological activity of a new gene in vivo
  (e.g., ref. 1 below), or
• Validate your target by transferring in a gene (or shRNA or microRNA) that either triggers a disease state, or diminishes or reverses symptoms in an animal model of disease. This information can be used to choose a target for your small molecule discovery program or your biological discovery program. (e.g., ref. 2 below)