Code
1SHHU24N
Duration
01 November 2023 → 31 October 2027
Funding
Research Foundation - Flanders (FWO)
Promotor
Research disciplines
-
Medical and health sciences
- Molecular medicine
- Genetics
- Transcription and translation
- Ophthalmology
Keywords
Antisense oligonucleotide (ASO)
Upstream open reading frame (uORF)
Project description
Inherited retinal disease (IRD) is a major cause of early-onset vision loss for which gene therapy is entering the clinic. Since gene augmentation is limited to relatively small disease genes, antisense oligonucleotides (ASOs) are an emerging strategy due to their easy design, chemical synthesis, and high target specificity. However, current ASOs are mostly mutation-specific and therefore only applicable to very small patient cohorts, given the tremendous allelic heterogeneity that is typical for IRD. In this PhD project, I aim to develop a new ASO therapy increasing translation of genes modulating common IRD disease mechanisms in order to treat a larger IRD patient cohort.