New Investigative Therapy Holds Promise for Retinitis Pigmentosa in Mouse Model

Researchers from Nanoscope, thanks to funding by the NIH, have developed a protein that can effectively restore vision in mice. This study was published in Nature Gene Therapy.

The Study

This research team effectively developed a protein, named MCO1, that can sense light and ultimately restore vision when gene therapy is used to attach the protein to bipolar cells on the retina. Remarkably, this process was found to be successful in mice that were completely blind. The treated mice responded faster to visual tests than non-treated mice. These tests included mazes and the ability to detect motion changing.  

But, you may be wondering, how exactly does this protein work? In individuals suffering vision loss from macular degeneration, retinitis pigmentosa, and other conditions, photoreceptors are damaged. However, the bipolar cells in these patients are still functioning. The idea was to have the bipolar cells take on the role of the malfunctioning photoreceptors.

It is really quite simple. Since bipolar cells are located past photoreceptors in the retina, when researchers add the MCO1 opsin gene (which works as a signal), they are able to restore light sensitivity.

In the past, researchers have uncovered that gene therapy can treat conditions like Leber congenital amaurosis, where the photoreceptors are still intact. Bionic eyes are also an option for some conditions; however, the surgery is very invasive. Additionally, although opsin therapies have been studied before, they have required intense light in order for the signal to be adequate. Of course, we know that intense light to the eyes is not healthy and in fact, can cause increased damage.

MCO1 is unique because it doesn’t require a strong light in order to advance the signal. Additionally, researchers are hopeful that it could benefit a wide array of conditions.

Upcoming Clinical Trial

The team is planning a clinical trial for this therapy later in the year. In mice models, no safety issues were found.

The researchers believe that the therapy could produce 20/60 vision in patients, but a trial in humans is a necessary step in order to understand the full benefits. Besides basic sight, there are other tests that can be examined in a human patient. For example, can patients treated with this therapy make out moving objects as well as someone with healthy vision?

You can read more this investigative therapy and upcoming trial here.

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