Gene Therapy Holds Promise for Treating Retinal Disease: Study

Wed May 10 2023
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ISLAMABAD: A groundbreaking study conducted by experts in Canada has revealed the potential of gene therapy in treating degenerative retinal disease, offering hope to millions of people worldwide who suffer from vision loss due to the death of retinal receptors.

The researchers focused on dormant support neurons called Muller glial cells, which exist in the human eye. They discovered that these cells can be transformed into tissues that function as cone photoreceptors, essential for visual acuity.

While this process has only been tested on mice so far, scientists believe it could be developed into a therapy that restores vision in humans.

Camille Boudreau-Pinsonneault, a neuroscientist and the first author of the study from the University of Montreal, explained that while Muller glial cells can reactivate and regenerate the retina in fish, they typically do not do so in mammals, including humans, after injury or disease. The researchers aim to understand why this difference exists.

Study focused on two genes

The study focused on two genes, Ikzf1 and Ikzf4, and their produced proteins, which are responsible for forming cells into different types.

By isolating and culturing the Muller glial cells before reprogramming them with temporal identity factors, including Ikzf1 and Ikzf4, the researchers were able to partially transform these cells into cone photoreceptor-like cells.

While glial cells normally provide nourishment and support to other cells in the eye, the surplus of these cells allows for their safe conversion into photoreceptor-like cells. These cells are crucial for perceiving light and discerning colors.

Although the trial is still in its early stages, the potential for this process to be replicated in humans without the need for cell transplantation is promising.

Moreover, the research findings could have implications for treating brain diseases by reprogramming damaged neurons with other cell types.

Ajay David, a doctoral student from the University of Montreal, expressed optimism about the study’s implications, stating that one day it may be possible to stimulate the cells naturally present in the retina to regenerate lost retinal cells and restore vision in individuals affected by pathological conditions.

While further research is needed to refine the cell transformation process and improve its efficiency, the study represents a significant step forward in the quest to develop effective treatments for retinal diseases and potentially other neurological conditions.

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