Targeting genes to specific neuronal or glial cell types is essential for developing safe and effective gene therapies for the eye and brain disorders. Adeno-associated viral vectors (AAVs) are the most promising tools for retinal and brain gene therapy, but targeting expression to specific cell types is still a challenge.

The Complex viruses platform designs different synthetic promoter/enhancer sequences to be embedded into the AAV genome using various strategies. The platform screens the generated viral vectors for cell type specific expression in human retinal organoids and in human retinas, focusing on resources and methods that provide economic, fast, and efficient cell-type targeting in a variety of species, both for fundamental science and for the development of cell-type-specific therapies for blinding diseases.