Gene tech for eye issues
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Australian scientists have made a breakthrough in gene therapy for eye diseases.
Researchers have successfully tested a novel gene therapy technique that may offer a future alternative to frequent eye injections for patients with vision-impairing conditions.
The Centre for Eye Research Australia (CERA) says its new research reveals the potential of gene therapy to treat a major cause of vision loss linked to ‘wet’ age-related macular degeneration and diabetic eye disease.
Led by Satheesh Kumar and Associate Professor Guei-Sheung Liu, CERA's preclinical study employed CRISPR Cas13 - a specialised RNA-editing tool - to suppress vascular endothelial growth factor (VEGF) production in retinal cells.
VEGF, a protein associated with the abnormal growth of blood vessels in the retina, plays a significant role in conditions causing vision loss in more than 200 million people globally.
“Our study shows the potential of RNA editing to develop gene therapies that offer an alternative treatment to the invasive, frequent eye injections that are currently used to treat wet macular degeneration and diabetic eye disease,” said Associate Professor Liu.
The team, including scientists from CERA, the University of Melbourne, and other institutions, adapted CRISPR Cas13 technology to modify the VEGF-producing mRNA in retinal cells, rather than the permanent genetic code.
This approach, tested in both mouse models and retinal cells derived from human stem cells, demonstrated effective reduction of VEGF, potentially slowing disease progression.
RNA editing offers an advantage by allowing for non-permanent adjustments to the genetic instructions that control cellular behaviour.
“This could allow treatments to be adjusted over time, depending on clinical need,” said Kumar.
The technique could treat degenerative eye conditions that otherwise require regular injections.
Currently, people with conditions like macular degeneration may require injections every six to 12 weeks, which, according to Associate Professor Liu, is costly and disruptive to quality of life.
“We envision that RNA editing could become a viable alternative to invasive and costly eye injections that have become a fact of life for many people living with wet macular degeneration or diabetic eye disease,” he said.
Though in its early stages, this discovery presents a promising step towards potentially transforming eye disease treatments through precise and customisable gene therapies.
More development is needed before clinical trials can commence.
