Gene therapy of the optic nerve
The optic nerve was restored for the first time with the help of gene therapy
To do this, scientists used the protein protrudin
Molecular biologists have figured out how to stimulate the restoration of the optic nerve, through which information about what our eyes see gets into the brain. Scientists tested the created gene therapy in front of several rats. An article with the results of the study was published by the scientific journal Nature Communications (Petrova et al., Protrudin functions from the endoplasmic reticulum to support axon regeneration in the adult CNS).
"With the help of gene therapy, we delivered the protein protrudin to the damaged part of the nerve. In the future, it will be possible to protect retinal neurons from death in a similar way, as well as restore them. To do this, however, it is necessary to conduct many more experiments and understand whether this therapy will work inside the human body," she said Veselina Petrova, a molecular biologist from the University of Cambridge (UK) and one of the authors of the study.
The eyes of humans and all other mammals contain several types of nerve cells. Some of them, such as cones and rods, are responsible for converting light particles into electrical impulses understandable to the brain. Other cells, including the optic nerve, are involved in transmitting these signals to the vision centers of the brain. If the work of both cells is disrupted, a person may develop various forms of blindness.
Scientists are trying to correct such defects both with the help of stem cells, which can turn into "blanks" of retinal cells, and gene therapy, which "reprograms" some of the surviving cells, forcing them to grow, turn into receptors or perform other functions. For example, four years ago, geneticists from the USA "repaired" the DNA in the retinal cells of mice using the CRISPR-Cas9 genomic editor, protecting them from complete loss of vision.
On the way to full recovery
In a new study, Petrova and her colleagues studied how the behavior of nerve cells will change if they are forced to produce large amounts of the protein protrudin. Scientists have recently found out that it stimulates the increase of various kinds of outgrowths in all other types of cells, except neurons, where its concentration is almost always low.
The researchers tested what would happen if they injected a lot of protrudin into neurons or forced these cells to produce a similar protein. For these experiments, Petrova and her colleagues created a gene therapy based on adenovirus, which causes the cell to produce molecules of this peptide, and then infected the culture of neurons with such adenoviruses.
Experiments have confirmed that protrudin stimulates the growth of axons – endings that connect neurons with neighboring nerve cells or other tissues of the body. Having made sure of this, the scientists traced how gene therapy acts on the damaged optic nerve and retina of several rats.
It turned out that this protein really accelerates the healing of the damaged nerve, and also protects the retina from minor damage. Scientists suggest that a similar method can be used to build up nerve endings in other parts of the nervous system, which opens the way for the widest use of protrudin in medical practice.
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