Gene therapy products animal models

The utilization of relevant animal species/models that can mimic the human physiology as closely as possible is critical in order to provide an activity and a safety profile for the gene therapy product of interest. This way measurable boundaries can be obtained for an acceptable risk-benefit ratio that will allow for the administration of the gene therapy product into the desired human population (i.e., bench to bedside translation Figure 32.9). There should be a... 

Validation of viral clearance is a major concern for products derived from mammalian cell culture and transgenic animals, as well as for viral vectors used for gene therapies. As we learn more and more about potential risks from newly found viruses, the requirements for validation increase. The increased concerns may be reflected in the number and types of viruses that are used for viral clearance studies. Both relevant and model viruses are used. A recent review of validation of the purification process for viral clearance evaluation provides further information on selection of viruses and performance of the studies [36],... 

Now that the gene and gene product of CF have been identified, gene therapy becomes an obvious potential for treatment. Research to date has centered on introduction of the correct gene into affected tissues. Viral vectors, chiefly adenovirus, have been studied in animal models, and human trials are under way. Liposomes may represent another useful delivery mode to introduce the correct gene. 

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