StarT · European Training Network to Diagnose, Understand and Treat Stargardt Disease, a Frequent Inherited Blinding Disorder

StarT will create an interdisciplinary and intersectorial European training network focusing on different aspects of autosomal recessive Stargardt disease (STGD1), a frequent inherited blinding disorder that affects an estimated 925,000 persons worldwide. StarT research aims to uncover the regulation of its disease gene ABCA4 and its missing heritability, in order to develop novel treatments. StarT training will give young researchers unparalleled training opportunities in outstanding vision research groups with unique expertise in omics technologies, bio-informatics, stem cell biology, animal models of disease, and therapeutics, providing each ESR with the necessary competences in state-of-the-art academic and industrial research. STGD1 is due to ABCA4 mutations, however up to 35% of STGD1 cases carries one or no ABCA4 coding mutation. New unconventional classes of ABCA4 mutations were recently discovered by us, the significance of which largely remains elusive. In order to understand the mechanisms triggered by these missing ABCA4 mutations and to design new therapies for STGD1 cases, challenging research questions will be addressed by the integration of unique skills from this network. Early-Stage Researchers will perform cutting edge research using innovative and interdisciplinary approaches: (functional) genomics and transcriptomics, bio-informatics, CRISPR/Cas9 genome editing, generation of stem cell and animal disease models and design of new treatments. The training objectives will be met through academic and industrial training-by-research via individual research projects, secondments, and network-wide training sessions.