Targeting RNA as an approach for treating retinal disease
Today, vision impairment up to total blindness represents an increasing global problem
This problem leads to a significant social and economic impact on affected patients and society as a whole.
Despite the advancing of knowledge on their genetic basis, there is no effective cure for retinal diseases to date and therefore there is an urgent need to fill this gap. Funded by the Marie Skłodowska-Curie Actions programme, the RETORNA project aims to find treatment for RD based on RNA therapy.
Research objectives
The main scientific aim is to observe changes in miRNA/mRNA profile of affected cells in retinal degenerations and correct those using different molecules, such as miRNA mimics, RNA-aptamers, and antisense oligonucletides.
Screening/profiling miRNA and mRNA in in vitro models of RD (ARPE19 cells, organoids)
Profiling miRNA and mRNA in animal models of RD and in human patients
Developing in silico models and computer tools to recognize molecular targets
Target modified RNA in vitro and observe results
Treat animal models (in vivo) and retinal organoid models with selected molecules to confirm in vitro results
Study the economic possibilities of a potential RNA therapy for RD
RETORNA in numbers
Project starting date
Duration
The objective is to create RNA profiles for different RD and identify several potential targets that will be used as therapeutic approaches.
Countries involved:
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