Therapy and RNA Group GhenT (TaRGeT)
Head
Research
Mission
TaRGeT aims to identify novel non-coding targets for gene therapy, shifting focus from DNA to RNA cis-regulatory elements. We are particularly interested in the role of long non-coding RNA (lncRNA) and cis-regulatory elements located in the 5’ untranslated region (5’UTR). Given the often tissue- and even species-specific function of these elements, we make use of human retinal models including adult post-mortem retina and retinal organoids for identification and functional validation. In a second research line, we aim to develop new antisense oligonucleotides (ASOs) directed against both coding and non-coding targets. TaRGeT investigates various ASO mechanisms, including RNAse-H1 based mRNA degradation and splice modulation.
Interests
- Development of a novel platform for antisense oligonucleotide therapy
- Focus on rare ophthalmic diseases
- Partner of the RARE-MED consortium, that aims to address missing heritability in rare disorders