DFG Research Unit for Ocular Gene Therapy - FOR5621

Adressing the unmet needs in ocular gene therapy
Sight is our most valued sense. However, 2.2 billion people globally suffer from vision impairment. Age-related macular degeneration (AMD), diabetic retinopathy (DR) and inherited retinal disorders (IRDs) are among the most common causes of irreversible vision impairment and blindness.
Over the past decades, major achievements in gene manipulation enabled new treatments.
They rely on adeno-associated virus (AAV) vectors to supplement a healthy copy of the disease-causing gene into a patient’s cells. While this concept is promising, it is limited by the unability to target recessive diseases and the cargo capacity of AAVs.
To overcome these challenges, the FOR5621 research unit is investigating novel technologies in ocular gene therapy (OCU-GT) .We work on improving viral vectors and disease models, better imaging and analytical methods, novel gene therapy modalities and machine learning approaches.