Bl6 Ndp-KO systemic gene therapy whole cochlea samples
We aimed to identify the effect of an AAV-based NDP gene therapy for Norrie disease. This therapy was tested on an Ndp-KO mouse model previously shown to  recapitulate Norrie cochlear phenotype (Bryant et al 2022, PMID 35132964).  We used transcriptomic analysis of whole cochlea lysates to determine the effect of this therapy on pathology related genes and downstream targets of Norrin signalling. Mice were treated at postnatal day 2 and cochleas collected for analysis at 2 months old.