Targeting cell types with gene therapy vectors

Targeting genes to specific neuronal or glial cell types could be essential in making gene therapies for the eye, or indeed for other neural systems in the brain or elsewhere. Adeno-associated viral vectors (AAVs) are the most promising approach for delivering gene therapy into the retina or the brain, but targeting expression to specific cell types is a challenge. In our research, we are designing different synthetic promoter/enhancer sequences to be embedded into the AAV genome using various strategies. We are screening the generated viral vectors for cell type specific expression. Using cell-type specific AAVs, we aim at demonstrating applications for recording, stimulation and molecular characterization of targeted cells. We are focusing our research 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 gene therapy of cell-type-specific human blinding diseases.

Please click here for a pdf excerpt of the IOB Annual Report 2018. It explains what the complex viruses platform is working on.

For the full version of the IOB Annual Report 2018 please click here.

Group Leader: Josephine Jüttner

Technical/Research associates

• Philipp Timo Kleindienst
• Jannick Imbach
• Lisa Locher
• Adrienn Volak