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Distinguished Speaker Series | Bence Gyorgy MD, PhD

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GHEI building, UCI
3d floor, Cavanaugh Room A & B

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The Center for Translational Vision Research Distinguished Speaker Series, also known as "Friday Seminars" showcases innovative research across the world. The seminar series has now been expanded to include lectures by experts on topics ranging from Ophthalmology, Genetics, Biochemistry, Neurobiology, Imaging, Computational Sciences to Novel Ophthalmic Treatments.

October 6, 2023 | Bence Gyorgy MD, PhD

Adenine base editing for gene correction in Stargardt disease

 

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Bence Gyorgy  MD, PhD
Bence Gyorgy MD, PhD
  • Head of the Ophthalmic Translational Research Group at the Institute of Molecular and Clinical Ophthalmology Basel (IOB)

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 allow us to test gene therapy in vitro in disease models resembling the human retina.

Our 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.

We act as a bridge between IOB’s Molecular and Clinical Center as well as our patients to allow rapid translation of scientific results into therapy.

Current projects:

Base Editing for Stargardt Disease

Optogenetics for Early-Onset Severe Retinal Dystrophy

Prime Editing for Usher Syndrome Type 2A