Stylianos Michalakis

Stylianos Michalakis is a Professor of Ocular Gene Therapy at the Department of Ophthalmology, Medical Faculty of Ludwig Maximilian University of Munich (LMU). He serves as a GSN full member and regular member of MCN, leading cutting-edge research in retinal gene therapy. His laboratory focuses on developing adeno-associated virus (AAV) vector-based treatments for inherited retinal diseases, with emphasis on cyclic nucleotide-gated channel disorders.

His research spans gene therapy vectorology, epigenetic regulation (particularly TET enzyme mechanisms), and neuroscience of retinal degeneration. Utilizing iPSC-derived retinal organoids, mouse models, and advanced molecular techniques, his work addresses unmet needs in treating conditions like retinitis pigmentosa and achromatopsia. The group employs CRISPR/Cas systems, omics technologies, and functional retinal analysis.

Michalakis co-founded the RD-CURE consortium, which pioneered the world's first gene therapy trial for CNGA3-linked achromatopsia and completed the initial trial for PDE6A-linked RP. His recent publications reveal a strong trend toward AAV capsid engineering for improved retinal targeting, long-term efficacy validation in large animal models, and novel diagnostic platforms using CRISPR activation. Key themes include choroidal delivery optimization, microglial interactions, and epigenetic mechanisms in retinal function.

Scientific recognition includes:

• Named inventor of two granted gene therapy patents and six patent applications
• Leadership in the NIH-funded R24 translational program for CNGB1-linked RP
• World's first ocular gene therapy trial for CNGA3 achromatopsia

He mentors current students Grazia Giorgio, Anna Winkler, and Sabrina Babutzka, while supervising notable graduates including Dr. Mirja Koch and Dr. Anna Sophie Geserich. His research receives substantial grant support, particularly through the RD-CURE consortium and NIH collaborations, focusing on translating vectorology breakthroughs into clinical applications. The laboratory maintains state-of-the-art facilities including confocal microscopy and retinal functional analysis systems.

Current initiatives involve developing novel AAV variants for intravitreal delivery, investigating TET3's role in retinal development, and advancing dual-vector systems for large-gene disorders like Stargardt disease.