A new molecular tool for treatment of age-related visual deterioration

2022.01.31 | Graduate School of Health

On Friday 11 February at 13.00, Sidsel Alsing Kalaee defends her PhD dissertation entitled " RNAi-Based Gene Therapy for Retinal Diseases".   

Age-related visual deterioration may be caused by neovascularization in age-related macular degeneration (AMD) which is common among the elderly. The current treatments require repeated administrations and not all patients benefit from them. Gene therapy offers the possibility of achieving life-long effects after only a single injection. In this PhD project from Aarhus University, Health, molecular tools for a gene therapy-based treatment for AMD are investigated. The tools are based on small regulatory RNAs which are designed to reduce the level of vascular endothelial growth factor (VEGF) which is pathologically elevated in AMD, and which drives the neovascularization. The RNA molecules are delivered to the cells in the eye using a viral vector. In this project, the technology is investigated in several different cell models as well as in a mouse model. We have developed specialized regulatory RNA molecules which are highly efficacious and have a favorable safety profile. The results show that the RNA molecules reduce the level of VEGF and the development of neovascularization. The RNA molecules have been shown to be expressed in the retina up to 2 months after treatment. Furthermore, we have shown the the RNA molecules can be delivered alongside a therapeutic protein factor to obtain a superior therapeutic effect. Thus, the project provides a foundation for development of efficacious and safe gene therapy for AMD.

The summary is written by the PhD student.

The defence is public and takes place in the Eduard Biermann auditorium, Aarhus University, Bartholins Allé 3, 8000 Aarhus C. Please see attached press release for more information.

Contact

PhD student Sidsel Alsing Kalaee

Mail: sidselalsing@gmail.com 

Phone: 61682068

Read full press release