Study Suggests that Gene Therapy Could Protect Multiple Sclerosis Patients from Vision Loss

According to a story from Medical Xpress, a team of researchers from the medical school at the University of Massachusetts has revealed new information about the nature of neurodegenerative symptoms in multiple sclerosis. The study also demonstrated how gene therapy could potentially protect patients from experiencing vision problems and loss. The research was led by Dorothy Schafer, Ph.D.

About Multiple Sclerosis

Multiple sclerosis is a neurological disease which is characterized by damage to the myelin sheath, a fatty, insulating, protective covering that surrounds nerve cells and allows them to communicate effectively. Although a precise cause has not been determined, multiple sclerosis is considered an autoimmune disease, in which a certain trigger, such as an infection, may cause the immune system to mistakenly attack healthy tissue. Smoking and certain genetic variants are also considered risk factors for the disease. Symptoms include blurred vision, double vision, blindness in one eye, numbness, abnormal sensations, pain, muscle weakness, muscle spasms, difficulty speaking and swallowing, mood instability, depression, loss of coordination, and fatigue. There are a number of treatments available for the disease, but no cure. Life expectancy for patients is slightly reduced. To learn more about multiple sclerosis, click here.

Study Results

The researchers describe in detail synapses are damaged in the disease through molecular processes. The data from the study suggests the possibility of developing a method of treatment that prevents synapses from being damaged; this could potentially be applicable to a variety of neurodegenerative diseases beyond just multiple sclerosis. Dr. Schafer says that a lot of research related to the mechanism of the illness has focused on the death of axons and damage to the myelin sheath, but the impacts in synapses may be just as important.

Vision loss is one of the most life-limiting and debilitating effects of this disease. In the models investigated, synapse loss was the result of microglia, a type of immune cell, eliminating critical presynaptic linkages. A protein called C3 was also detected at the synapses, which is typically associated with brain development and is normally not present in the brains of adults.

Using a mouse model, the scientists used an adeno-associated viral vector to deliver a natural inhibitor of C3 called Crry. The approach appeared successful, and the affected mice saw improvements to their vision. While further research is necessary in order to understand the presence of C3, the results nevertheless point to the capability of treatments that target neural circuits in the brain to protect against neurodegeneration.

Check out the original study here.


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