Gene therapy restored nerve connections in the retina and improved vision

Gene therapy has made it possible to restore damaged nerve connections in the retina of an adult eye and improve vision. Scientists from Michigan State University have shown that the mature retina is able to remodel itself even after long-term disturbances. The study results were published in the journal Molecular Therapy Advances (MTA).

Researchers studied an inherited vision disorder caused by a defect in the CaBP4 gene. This gene is responsible for the production of a protein necessary for transmitting signals from light-sensitive cells in the retina to other nerve cells. Due to the mutation, this process is disrupted, so vision problems arise already in childhood.

The experiment was carried out on dogs with this mutation. A safe viral vector with a working copy of the CaBP4 gene was injected into the retina of animals once. After treatment, vision improved significantly, especially in low light.

At the same time, the therapy did not just stop further deterioration of vision. Scientists found that the retina began to restore the structures necessary for the transmission of visual signals, and pre-existing disorders were partially corrected. The effect lasted up to three years of follow-up.

According to the researchers, the results show that the adult retina has a greater ability to restore neural connections than previously thought. In the future, this mechanism may help in the development of methods for treating hereditary vision diseases.