Barriers and carriers for non-viral delivery of mRNA to induce transient protein expression in the retina: the hurdles of the inner limiting membrane and innate immunity versus the potential of mRNA self-amplification.

01 October 2019 → 01 November 2019
Regional and community funding: Special Research Fund
Research disciplines
  • Medical and health sciences
    • Nucleic acids
    • Ophtalmology
    • Biopharmaceutics
mRNA self-amplifying mRNA inner limiting membrane non-viral gene delivery intravitreal injection (lipid) nanoparticles
Project description

The intravitreal delivery of mRNA packaged into nanoparticles to reach the inner retina is mainly hindered by the inner limiting membrane and the innate immunity that detects and destroys mRNA. Therefore, several goals were set up: (1) design small nanoparticles (<80 nm), (2) evaluation of selfamplifying
mRNA, (3) mRNA combined with innate immunity inhibitors and (4) mRNA vs. Selfamplifying mRNA.