Gene & Cell Therapy Case Study

Optimizing in vivo gene delivery with rAAV

The humanized FRG KO model allows delivery optimization of payloads.  In the example below, humanized FRG KO mice were transduced with 2 X 1011 or 8 X 1011 genome copies of a recombinant AAV-LK03:eGFP. At 13 days post-transduction the livers were isolated and three independent images of each genome dose were captured using an EVOS fluorescent microscope.  The images validate the ability to quantitate delivery of payload in a dose dependent manner.