Australian researchers have used a gene therapy technique to suppress the production of vascular endothelial growth factor (VEGF) in human retinal cells.
The pre-clinical research, published in the Proceedings of the National Academy of Sciences,1 was led by Satheesh Kumar and Associate Professor Guei-Sheung (Rick) Liu from the Centre for Eye Research Australia (CERA) and University of Melbourne, along with scientists from the University of Sydney, Children’s Medical Research Institute, University of Western Australia, and Zhongshan City People’s Hospital, China.
The researchers for the first time used an RNA editing tool – known as CRISPR Cas13 – to suppress the production of VEGF, a protein that is the key driver of vision loss in diseases such as neovascular age-related macular degeneration and diabetic macular oedema.
… the new research demonstrated the potential to develop a gene therapy… as an alternative to current treatment delivered by invasive eye injections
The experiment targeted the mRNA sequence that instructs cells to produce VEGF. It delivered the RNA editing tool via an AAV viral vector and was tested on a mouse model and human retinal cells derived from stem cells. It showed that the viral vector was effective in delivering the treatment to retinal cells and produced a significant reduction in VEGF, and a slowing of disease progression in the mouse model.
Assoc Prof Liu said the new research demonstrated the potential to develop a gene therapy using CRISPR Cas13 to control VEGF as an alternative to current treatment delivered by invasive eye injections.
Reference
- Kumar S, Hsiao YW, Liu GS, et al. Characterisation of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina. Proc Natl Acad Sci U S A. 2024 Nov 5;121(45):e2408345121. doi: 10.1073/pnas.2408345121. Epub 2024 Oct 30.
