Gene Therapy

Newcells’ retinal organoids have shown their potential to be used as an informative in vitro model for the evaluation of new retinal gene therapy vectors for retinal diseases.

Our organoids have been used to screen AAV gene therapy vectors in a collaboration with a team at the University of Oxford. This study confirmed robust and efficient transduction of human photoreceptor-like cells by AAV vectors highlighting improved transduction capability of AAV2 7m8 in retinal organoids when using the ubiquitous CAG promoter. With several cell types present within the organoids, the experiments were able to specifically show the targeted cell types, demonstrating that a CAG-driven transgene transduced a broad range of cell types, while GRK1-driven transgenes show a more restricted photoreceptor-specific expression (A). The work also demonstrated the viability of the retinal organoids were not affected by AAV transduction (B).

These studies can be carried out in both our retinal organoids and RPE in vitro models to allow rapid evaluation of new retinal gene therapy vectors.

Newcells provides a custom service evaluating AAV vectors in our laboratories. With our regular supply of tissue, our projects timelines are short. The robust data generated by our scientific experts will guide you in confidence for key decision-making steps during drug development.

An example of viral vector testing service includes a set of assays to assess transduction efficiency, cell tropism and post-transduction cell viability.