Design of a novel antisense oligonucleotide therapy for inherited blindness

01 November 2020 → 31 October 2021
Regional and community funding: Special Research Fund
Research disciplines
  • Medical and health sciences
    • Transcription and translation
    • In vitro testing
    • Genetics
    • Ophtalmology
  • Engineering and technology
    • Gene and molecular therapy
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.