Potential: Macquarie reseachers say there is optimism gene therapy will be a valuable addition to other treatments that make eye nerves more resistant to the mechanisms that cause glaucoma.
Macquarie Medical School researchers have developed a technique for a gene therapy that could help treat the world’s leading cause of irreversible blindness.
A treatment that ensures nerve cells in the eye continue to produce a vital protein that protects them from being broken down could help prevent the progression of glaucoma, researchers have found.
Glaucoma is the world’s leading cause of irreversible blindness, affecting about 300,000 Australians and more than 70 million people globally. It is associated with gradual vison loss, initially in the periphery but then spreading centrally. The disease damages the optic nerve and the retinal ganglion cells, which are a type of neuron that carry visual information to the brain.
Glaucoma is often associated with increased pressure in the eye, and it can be detected during routine vision tests, by measuring the eye pressure or inspecting the optic nerve for damage with special scanners.
While any damage already sustained cannot be reversed, in many cases the progression of the disease can be slowed or stopped through treatment to lower the pressure.
However, many patients continue to worsen despite treatment, indicating there are other factors at play in addition to pressure.
Professor of Ophthalmology Stuart Graham from Macquarie Medical School is leading a team that is investigating the role of the protein neuroserpin in the disease.
A first
Vision Neurobiologist Associate Professor Vivek Gupta says they have found that neuroserpin, which is produced in the connectors between nerve cells, is vital in protecting retinal ganglion cells.
“Other researchers have linked changes in neuroserpin to stroke and neurodegenerative disorders like Alzheimer’s and Parkinson’s diseases, but our work is the first to relate it to glaucoma,” says Professor Gupta.
“Cells naturally break down and are recycled in the body, but when neuroserpin is absent, this process speeds up in the retina.
“Essentially, the body begins to eat away at the retinal ganglion cells and the optic nerve.”
Oxidation is a common cause of molecular breakdown in nature. Iron rusting and a cut apple turning brown are both familiar examples.
The team has discovered that when neuroserpin oxidises, it loses its protective ability, allowing accelerated cell breakdown.
They have also shown that when mice produce more neuroserpin, it has a protective effect, promoting the survival of the retinal ganglion cells and minimising glaucoma damage.
Vision Scientist Dr Nitin Chitranshi says in the latest work, detailed in an article in the latest edition of Molecular Therapy, the team has successfully manipulated a gene in mice to produce a version of neuroserpin that is resistant to oxidation.
“When we introduce this gene directly to the eye, it increases the production of neuroserpin in the retina,” says Dr Chitranshi.
“We are also working on a way for the protein to give its instructions to produce neuroserpin only to the retinal ganglion cells and not to other neurons, so it can be perfectly targeted.”
The team is now preparing for further testing of the enhanced gene and will commence new trials shortly.
Professor Graham says glaucoma is a complex disorder involving a number of mechanisms, not all of which are well understood.
“For this reason, our gene therapy is unlikely to be a silver bullet for all glaucoma, but we have great hopes that it will become a valuable part of treatment for use in conjunction with other therapies, making the nerve cells more resistant to damage,” Professor Graham says.
“But we still have a lot of work to do before we can translate this to human studies.”
Original Article: Retinal gene therapy could provide protection in glaucoma
More from: Macquarie University
The Latest Updates from Bing News
Go deeper with Bing News on:
Glaucoma gene therapy
- BrightFocus Foundation Announces $10M in New Funding Across Brain and Vision Research, Celebrates Historic Diversity of Grant Award Recipients
including the first commercially available blood test in the U.S. to identify early signs of Alzheimer’s disease and the first-ever reversal of age-related vision loss from glaucoma using gene therapy ...
- CRISPR Therapeutics to Present Oral Presentation at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting
ZUG, Switzerland and BOSTON, April 22, 2024 (GLOBE NEWSWIRE) -- (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today ...
- Macular Degeneration vs. Glaucoma
Vision loss due to dry AMD is permanent, while wet AMD can be slowed and sometimes reversed with medications and laser therapy. Glaucoma is usually treated with eye drops or surgery to relieve ...
- Is cell and gene therapy the future of medicine?
Brian Harrison of Hitech Health believes cell and gene therapy treats 'the root cause' of diseases, but challenges exist in the sector.
- AAV-mediated gene therapies for glaucoma and uveitis: are we there yet?
Gene therapy, particularly utilising adeno-associated virus (AAV ... This review discusses strategies employed in AAV-based gene therapies for glaucoma and non-infectious uveitis and provides an ...
Go deeper with Bing News on:
Glaucoma
- Glaucoma’s figures call for decisive action
Diseases are bad news, and when one has gained notoriety like the way glaucoma is prevalent in Ghana, it calls for a lot of concern. Glaucoma remains one of the primary causes of blindness in the ...
- ASCRS 2024: Third-generation trabecular micro-bypass in eyes failing prior glaucoma therapy
Deborah Gess Ristvedt, DO, shares highlights from her presentation and what these real-world cases mean for clinicians and their patients with glaucoma.
- Contact Lenses Market, Projected to Reach US$ 16,256.8 Million by 2034: A Detailed Analysis of Growth Factors, Consumer Trends
In 2024, the global contact lenses market is estimated to be valued at US$ 10,035.9 million, with projections indicating a growth to US$ 16,256.8 million by 2034. Over the forecast period, the demand ...
- BrightFocus Foundation Announces $10M in New Funding Across Brain and Vision Research, Celebrates Historic Diversity of Grant Award Recipients
Global nonprofit BrightFocus Foundation announces $10 million in new Alzheimer’s disease, macular degeneration, and glaucoma research grant funding—age-related diseases with no cure that affect more ...
- Glaucoma care, fraught with many challenges, requires multifaceted approach — Expert
An ophthalmologist, Professor Affiong Ibanga, has said that glaucoma care in Nigeria is fraught with many challenges and requires a multifaceted approach to ensure respite from glaucoma, the leading ...