Code
01D29520
Duration
01 November 2020 → 31 October 2021
Funding
Regional and community funding: Special Research Fund
Promotor
Fellow
Research disciplines
-
Medical and health sciences
- Transcription and translation
- In vitro testing
- Genetics
- Ophthalmology
-
Engineering and technology
- Gene and molecular therapy
Keywords
Inherited retinal dystrophies
gene therapy
antisense oligonucleotides
Project description
Inherited retinal diseases are a major cause of vision loss for which precision medicine is entering the clinic. Emerging antisense oligonucleotide (ASO) therapies are mostly mutation-specific, requiring many individual clinical trials. Here, we aim to design an innovative, mutation-independent ASO strategy targeting upstream open reading frames in order to increase protein translation, which is potentially applicable for many inherited diseases.