Preclinical testing of AAV9-PHP.B for transgene expression in the non-human primate cochlea.

We describe a transmastoid surgical approach needed to access the RWM of this common primate model. We found that AAV9-PHP.B transduced nearly 100% of both IHCs and OHCs, from base to apex, at the higher doses (3.5 × 1011 and 7 × 1011 vector genomes). However, at lower doses there was a steep reduction in viral transduction. Thus, AAV9-PHP.B efficiently transduces the IHCs and OHCs of nonhuman primates, and should be considered as an AAV capsid for inner ear gene therapy in humans.
PMID: 32145977 [PubMed - as supplied by publisher]

Source: Hearing Research - February 25, 2020 Category: Audiology Authors: Ivanchenko MV, Hanlon KS, Devine MK, Tenneson K, Emond F, Lafond JF, Kenna MA, Corey DP, Maguire CA Tags: Hear Res Source Type: research