Figure 6.

In Vitro Gene Therapy Testing Strategy for adBD

The amenability of adBD mutations to correction via gene augmentation can be evaluated for efficacy and safety in a dish using BEST1 mutant iPSC-RPE models. Those individuals with mutations that fail to respond to gene augmentation would then undergo further testing for gene editing (or another alternative strategy) using the same adBD iPSC-RPE model systems.