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Scientists at Trinity College Dublin have developed a new approach to gene therapy that offers one-day promise of treating an eye disease that leads to progressive vision loss and affects thousands of people around the world.
The study, which involved a collaboration with clinical teams at Royal Victoria Eye and Ear Hospital and Mater Hospital, also has implications for a much broader suite of neurological disorders associated with aging.
The scientists published their findings in the leading journal, Frontiers in Neuroscience.
Dominant optic atrophy (DOA)
Characterized by degeneration of the optic nerves, DOA typically begins to cause symptoms in patients in the early adulthood. These include moderate vision loss and some color vision defects, but the severity varies, symptoms can worsen over time, and some people can go blind. There is currently no way to prevent or cure DOA.
A gene (OPA1) provides instructions for making a protein found in cells and tissues throughout the body that is critical to maintaining the proper functioning of mitochondria, which are the energy producers in cells.
Without the protein produced by OPA1, mitochondrial function is not optimal and the mitochondrial network that is well interconnected in healthy cells is highly disrupted.
For those living with DOA, it is mutations in OPA1 and dysfunctional mitochondria that are responsible for the onset and progression of the disorder.
The new gene therapy
Scientists, led by Dr Daniel Maloney and Professor Jane Farrar of Trinity’s School of Genetics and Microbiology, developed a novel gene therapy, which successfully protected the visual function of mice that had been treated with a chemical that targeted mitochondria and consequently they live with mitochondria dysfunctions.
The scientists also found that their gene therapy improved mitochondrial performance in human cells that contained mutations in the OPA1 gene, offering hope that it could be effective in people.
Dr Maloney, researcher, said:
“We used an intelligent laboratory technique that allows scientists to deliver a specific gene to cells that need it using specially designed harmless viruses. This allowed us to directly alter the functioning of the mitochondria in the cells we treated, increasing their ability to produce energy which in turn helps protect them from cell damage.
“Interestingly, our results demonstrate that this OPA1-based gene therapy can potentially provide benefits for diseases such as DOA, which are due to OPA1 mutations, and perhaps also for a wider range of diseases involving mitochondrial dysfunction.”
Importantly, mitochondrial dysfunction causes problems in a number of other neurological disorders such as Alzheimer’s and Parkinson’s disease. The impacts gradually build up over time, which is why many may associate such ailments with aging.
Professor Farrar, research professor, added:
“We are very excited about the prospect of this new gene therapy strategy, although it is important to point out that there is still a long journey to complete from an R&D perspective before this therapeutic approach can one day be available as a treatment.
“OPA1 mutations are involved in DOA and therefore this OPA1-based therapeutic approach is relevant for DOA. However, mitochondrial dysfunction is implicated in many neurological disorders that collectively affect millions of people around the world. We believe there is great potential for this type of therapeutic strategy targeting mitochondrial dysfunction to provide benefits and thus have a major impact on society. Having worked with patients for many years living with visual and neurological disorders, it would be a privilege to play a role in a treatment that could one day help many. ”
(This story was published by a branch agency with no text changes. Only the title was changed.)
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