Project description
Research training network with a focus on hereditary blinding disorders
The ATP-binding cassette transporter gene (ABCA4) is the causal gene for Stargardt disease (STGD1), an inherited blinding disorder that affects an estimated 925 000 people worldwide. ABCA4 mutations are the most common cause of inherited retinal diseases leading to severe visual impairment and blindness. Funded by the Marie Skłodowska-Curie Actions programme, the StarT project is creating an interdisciplinary research training network focusing on all aspects of STGD1 to develop novel treatments. The early stage researchers in training will uncover the regulation of the ABCA4 gene and its missing heritability using innovative approaches, including functional genomics and transcriptomics, bioinformatics, genome editing, stem cells and animal disease models. The aim is to apply this knowledge to the design of new therapies.
Objective
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.
Fields of science
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- natural sciencesbiological sciencescell biology
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- natural sciencesbiological sciencesgeneticsmutation
- natural sciencesbiological sciencesgeneticsgenomes
Keywords
Programme(s)
Coordinator
9000 Gent
Belgium