HFA-Spaag

Hector Fellow Projekt Salome Spaag

01/08/2024 to 31/07/2028

Mutations in USH2A are one of the most common cause of Usher syndrome and Retinitis pigmentosa for which no cure exists so far. The project seeks to develop and validate a novel CRISPR technology based therapeutic approach targeting common mutations in the USH2A gene and to explore strategies for an inducible (switch-on/switch-off) transgene expression system implemented into an adenoviral-associated viral (AAV) vector for delivery into preclinical model systems.