Ophthalmic Translational Research Group
The Ophthalmic translation group focuses on translating science and technology to the clinic.
Recent advances in the understanding of genes, cell types and circuits involved in vision opened up the possibility to develop repair strategies, including gene therapy, for the loss of vision. Gene therapy for vision loss is now a clinical reality with the first approved product based on adeno-associated viral vectors. Today, gene editing nucleases and base editors allow us to manipulate genes with efficiency and precision we have never dreamed of. Light sensitivity of blind retinas can be restored using optogenetic sensors, and human retinal organoids allows one to test gene therapy in vitro in disease models resembling the human retina.
The group uses these novel technologies to modulate or take control over biological processes and bring them to patients in need as therapeutics. Clinical translation involves continuous interaction between basic researchers, technology specialists, clinicians, regulatory specialists, patient advocacy organizations and the patients themselves.
The group acts as a bridge between researchers and clinicians at IOB to allow rapid translation of scientific results into therapy.
Publications
High-efficiency base editing in the retina in primates and human tissues
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