Potential use of gene therapy to restore vision lost due to degenerative disease

A new method developed by Canadian scientists could potentially restore vision to individuals with inherited vision impairment. This regenerative therapy involves activating specific genes that can transform inactive cells in the retina into new light-sensing cells, which can replace the cells that were lost due to disease.

Retinitis pigmentosa is an example of genetic diseases that arise from the degradation of light-sensing cells in the retina. Over time, this impairs a patient’s vision, reducing their ability to detect details and colors, and creating a tunnel vision effect. In severe cases, it can ultimately lead to complete blindness.

The University of Montreal scientists have developed a technique to potentially reverse vision loss, according to a recent study. By activating “dormant” cells in the retina and reprogramming them into induced neuron cells, the team was able to convert them into new light-sensing cells that could restore lost vision.

The retina in fish can be regenerated by reactivating Müller cells, a type of glia that provides support to neurons in the retina, after it is damaged by injury or disease. Unfortunately, mammals lack this ability. However, a recent study by scientists at the University of Montreal screened a set of genes to identify any that could potentially unlock this regeneration process.

By conducting a screening of genes, the research team identified two transcription factors, Ikzf1 and Ikzf4, which can activate Müller cells in the retina and transform them into retinal neurons. This new approach could potentially help restore the function of light-sensing cells that have been lost due to retinal diseases. Similar studies using different transcription factors have also yielded promising results.

Although it is still in its early stages, the research provides hope for restoring vision to patients in advanced stages of degenerative diseases by regenerating the retina. Other treatments, such as gene therapies that prevent disease progression or implanting artificial retinas, have also shown promise.

Ajay David, the co-author of the study, stated that in the future, it might be possible to use the cells that are already present in the retina and activate them to regenerate light-sensing cells lost due to degenerative diseases, ultimately leading to the restoration of vision.

Source: University of Montreal