Developing Functional Analytical Assays for the Characterisation of Dual AAVs

Time: 1:30 pm
day: Track A Day 1 PM


  • The dual vector approach consists in dividing the gene of interest in two parts, one vector encoding the 5’ sequence and the other the 3’ sequence.
  •  In vitro bioassays to demonstrate the dual otoferlin vector expresses full-length human otoferlin mRNA and protein.
  • Determination of the optimal ratio vector for efficient reconstitution.
  • In vivo functional assays to show the recovery of auditory function