Current techniques for packaging AAV vectors are not readily amenable to large-scale production. These difficulties arise, in part, from toxicity of the AAV Rep proteins to helper viruses and host cells, thus requiring that the AAV rep/cap genes be provided on a separate vector from the helper virus or silenced in the cell chromosome. In addition, the Rep gene products are frequently cytotoxic to the cells used to package rAAV vectors. Temperature-sensitive AAV Rep proteins can be used to reduce or mitigate the problems posed by Rep protein toxicity. The activity of the temperature sensitive Rep proteins maybe readily controlled by shifting the cells packaging the rAAV vectors to permissive or non-permissive temperatures as desired.

File Number: 00-0001 

Other Information:

State of Development
pre-clinical