Hi Rick,
Sorry for the late recruitment, I thought I responded earlier, but I guess not...oops : )
This is an interesting method, but it will be tough as they need to get both vectors into the same cell for it to work...not to mention, will the splicing have an effect on the duration the vector stays in the nucleus? Also, they'll run out of diffent serotypes of AAV pretty quickly, when they need to repeat the treatment. In the discussion of the paper they mention that:
"In the mouse muscle, the trans-splicing vectors exhibited only 4.3% of the activity of the intact vector, and the overlapping vectors showed only 0.37% of the activity of the intact vector, suggesting that recombination is not a practical solution to increasing the genetic capacity of AAV vectors"
So I think that about summarizes the practicality of this method, unless it is more efficient in other tissues, or if they can some how tweak it.
BWDIK?
Best of luck,
SS |