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Our customer‘s goal was to confirm the dose-dependant ability of AAV constructs to transduce retinal tissue, more specifically the retinal pigment epithelium (RPE).
We followed our standardised series of steps to develop a customised solution.
We clearly defined the problem and questions to be answered through in-depth discussion between the customer and our technical experts.
The customer wished to rapidly evaluate if their AAV vector was able to transduce RPE cells and if the transduction efficiency was dose-dependant.
Taking all project specific factors into account, we suggested a two-step approach as being the most appropriate for their situation:
We designed the specific experiments and presented the plan in our Statement of Work.
Our experimental plan involved using fully validated human iPSC-derived RPE.
For Step 1: RPE transduction
For Step 2: Imaging analysis
Our scientists carried out the agreed experiments within 3 months providing regular updates to the customer.
The study included the experimental phase, data processing, data analysis and presentation of a data summary.
Example dataset
We delivered a detailed report with reliable dataset which was shared digitally and discussed over a call upon request.
The study clearly showed :
iPSC-derived RPE cells transduction study confirmed efficiency of AAV5 gene therapy vector to progress preclinical and in vivo studies.
We delivered a robust dataset which clearly showed that human IPSC-derived RPE cells are a robust in vitro model for testing of AAV constructs. Our analysis gave confidence to our customer to proceed to preclinical studies with their AAV construct.