Scientists Develop Gene Therapy for Eye Disease

Scientists from the Trinity College Dublin has just published a research article in a leading journal that shows promising result of a therapy to treat progressive loss of vision. A disease that affects thousands of people all over the world. Partnering with the Royal Victoria Eye and Ear Hospital and Mater Hospital, the study has found implications for a much wider suite of neurological disorder that is associated with ageing.

Dominant Optic Atrophy (DOA) is characterized by degeneration of the optic nerves which typically starts to cause various symptoms in early adult years of a patient. Symptoms includes moderate vision loss and some color vision defects which may vary from patient to patient but tend to get worse as they age. Sometimes it may even cause blindness which currently doesn't have any cure.

A gene which is known as OPA1 plays a crucial part for maintaining proper function of Mitochondria. Also known as the energy producers in cells which generally provides instructions to make a protein that is found in cells and tissues throughout the body. Without the protein made by OPA1, the mitochondrial function goes below sub-optimal level and the mitochondrial network gets highly disrupted.

A healthy cell should have well interconnected mitochondrial network as show in the picture provided below. Individuals with DOA tend to have mutated OPA1 and dysfunctional mitochondrial network which gradually causes the disorder in vision.

Healthy Cell Shows Mitochondrial Network
Healthy Cell Shows Mitochondrial Network in Gold Color. © Trinity College Dublin.

Scientists, led by Dr. Daniel Maloney and Professor Jane Farrar from Trinity's School of Genetics and Microbiology has initially treated a mice with a chemical that targeted the mitochondria and consequently was living with dysfunctional mitochondria. Later on, with their new gene therapy they were able to improve mitochondrial performance and protect the visual function of the mice.

Similar results were found in human cells that contained mutations of the OPA1 gene which the scientists believes could be an effective way to treat patients with DOA. Mitochondrial dysfunction causes a wide range of health issue including a suite of other neurological disorder such as Alzheimer's and Parkinson's disease. This newly developed Gene therapy shows huge potential on treating such diseases as well which may bring huge social impact.

The research was supported by Science Foundation Ireland, the Health Research Board of Ireland, Fighting Blindness Ireland, and the Health Research Charities Ireland.

Soruce: Trinity News, Research Article.


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