Novel Gene Therapy For Eye Disorders
Trinity College researchers, Dublin have designed a novel gene therapy method which provides a potential treatment possibility to an eye disease that ends up in progressive visual loss and impacts countless individuals around the world.
The joint study by the Mater Hospital and Royal Victoria Eye and Ear Hospital has wide-ranging applications for aging-associated neurological disorders.
The researchers published their findings in the journal, Frontiers in Neuroscience on 26th November 2020.
Dominant Optic Atrophy (DOA).
DOA is marked by optic nerve atrophy which commonly begins in early adulthood. Atrophy comprises of moderate blindness and few color vision impairments. With an increase in severity, the indications can deteriorate eventually. Up till now, there is no method or treatment for DOA prevention and cure.
All genes present a manual for cellular protein synthesis. The gene OPA1 plays a major role in regulating correct function in mitochondria, which is the powerhouse of the cell.
In the absence of the protein coded by OPA1, the performance of mitochondria is below par and the proper interlinking of healthy cells gets heavily distorted.
The onset and progression of DOA are attributed to mutations in the gene OPA1 and mitochondrial dysfunctions.
The Novel Gene Therapy
The novel approach study was supervised by Prof. Jane Farrar and Dr. Daniel Maloney, Trinity School of Genetics and Microbiology. They have devised a novel gene therapy, which proved successful in guarding rodent’s visual function after treatment with mitochondria-targeted chemical and were thus surviving with mitochondrial malfunctions.
The researchers additionally discovered that their novel gene therapy approach ameliorated the mitochondrial functions in OPA1 mutated human cells, giving hope for an effective treatment option.
Dr. Maloney, a research fellow, stated that they employed a shrewd lab procedure with notably manipulated non-harmful viruses to introduce certain genes to the cells. This facilitated them to directly change the mitochondrial working in the treated cells, enhancing their capacity to generate energy molecules to combat cell damages.
Stunningly, their results illustrated that the OPA1-based novel gene therapy can effectively offer advantages in treating disorders like DOA, which are caused by mutations in OPA1 genes and also perhaps for a large number of disorders related to mitochondrial malfunctions.
Significantly, malfunctioning mitochondria results in various other neurological diseases like Parkinson’s and Alzheimer’s. All these disorders worsen with time, which is why they are related to aging.
Prof. Farrar added that they are exhilarated by the future possibilities of this novel gene therapy approach, even though it takes considerable time for implementing the idea to therapy and accessible treatment.
Since OPA1 mutations are present in DOA, the OPA1-based treatment method is germane. Nonetheless, there are numerous individuals affected by neurological diseases associated with malfunctioning mitochondria. They believe that there is tremendous scope for this kind of treatment procedure aiming at mitochondrial malfunctions to offer an advantage and thus present a significant impact on society. It would be an honor to help the affected individuals by implementing this therapeutic strategy.
Gene Therapy For Eye Disorders
Author : Geema George