A new gene therapy against eye diseases


Last updated on December 8th, 2022 at 02:37 pm

Researchers at Trinity College, Dublin have just developed a new approach to gene therapy that offers the hope of one day treating dominant optic atrophy, an eye disease that causes progressive loss of vision.

Consisting of introducing genetic material to “correct” a patient’s cells, gene therapy has for several years offered promising results against serious and incurable diseases such as certain cancers and neurodegenerative diseases.

eye diseases
eye diseases


  • By restoring the functioning of the OPA1 gene, gene therapy has made it possible to improve the performance of the mitochondria, responsible for the appearance of dominant ocular atrophy.

  • This technique could also lead to treatments for other disorders linked to cellular aging, such as neurodegenerative diseases.

A new study published in the journal Frontiers in Neuroscience and conducted by scientists at Trinity College, Dubin, in collaboration with clinical teams from the Royal Victoria Eye and Ear Hospital and Mater Hospital, has shown that gene therapy could also lead to a treatment for dominant optic atrophy (DOA). More broadly, it could also have implications for a much wider range of neurological disorders associated with aging.

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Mitochondria degeneration

Characterized by degeneration of the optic nerves, the dominant optic atrophy usually begins to cause symptoms in patients in early adulthood: moderate vision loss and some color vision defects, although their severity varies. Symptoms can also worsen over time and lead to total blindness in some patients. There is currently no way to prevent or cure AOD.

Its occurrence is due to mutations in the OPA1 gene, essential for the proper functioning of the mitochondria, which are the energy producers in cells. Without the protein made by OPA1, mitochondrial function is suboptimal and the mitochondrial network, which is well interconnected in healthy cells, is severely disrupted, which can lead to the onset and then the progression of optic atrophy. dominant.


This new gene therapy has been successfully tested in mice treated with a chemical targeting the mitochondria and therefore living with dysfunctional mitochondria. It also improved the performance of mitochondria in human cells that contained mutations in the OPA1 gene, which gives hope that it could be effective in humans.

A potentially effective therapy against Alzheimer’s and Parkinson’s

The scientists also found that their gene therapy improved the performance of mitochondria in human cells that contained mutations in the OPA1 gene, raising hopes that it might be effective in humans.

“Our results fascinatingly demonstrate that this OPA1-based gene therapy has the potential to provide benefits for diseases like ODA, which are due to OPA1 mutations, and possibly for a wider range as well. of diseases involving mitochondrial dysfunction says Dr. Daniel Maloney, lead author of the study. This includes other neurodegenerative diseases like Parkinson’s and Alzheimer’s, which are linked to aging and the progressive dysfunction of mitochondria.

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“We are very enthusiastic about the idea of ​​this new gene therapy strategy” , explains Professor Jane Farrar, co-author of the work. Even if, she adds, “there is still a long way to go from the point of view of research and development before this therapeutic approach can one day be available as a treatment”.

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