Our researchers have published the results of a new study that sheds light on the treatment of retina dystrophies, a common health problem worldwide. Inherited retinal diseases (IRDs), which cause progressive vision loss, cannot be treated currently due to the inability to regenerate retina cells. This new study takes an important step forward in the treatment of IRDs with the use of RNA-based therapeutics called ASOs (anti-sense oligonucleotides).
The research aims to establish a reliable in vitro model using induced photoreceptor-like cells (PRCs) generated by direct programming from dermal fibroblasts. The study published in the Experimental Eye Research Journal. In this study, the efficacy of ASOs targeting the BEST1 gene was evaluated in this model. The results show that ASOs successfully degrade mRNA, specific to the targeted alleles with almost no off-target effects. These findings demonstrate the potential of ASOs as safe and effective therapeutic agents in the treatment of IRD.
This research is the first ASO-based in vitro study in Turkey on "RNA-based therapeutics". It is also emphasized in Hacettepe University 2022 - 2027 Research Priority Areas Report, which promises to open new horizons especially in the treatment of rare diseases. The results provide a valuable platform for developing personalized IRD therapies and inspire further research on RNA-based therapeutics. This achievement of Hacettepe University has been recorded as an important step in the scientific society and will contribute greatly to the development of future treatment methods.
The coordinator of the project is Assist. Prof. Beren Karaosmanoğlu from Faculty of Medicine, Department of Medical Genetics, the contributers are Assoc. Prof. Dr. Ekim Taşkıran, Instructor Gozde Imren from the same department, Prof. Dr. Hande Taylan Şekeroğlu from Department of Ophthalmology and Prof. Dr. Gülen Eda Utine from Department of Pediatric Genetics. The project was supported by Hacettepe University Scientific Research Projects Coordination Unit.
The link of the study: https://www.sciencedirect.com/science/article/pii/S001448352400054X?via%3Dihub