Wolfram Syndrome and its Degenerative Factors and Treatments

Olivia Tolbert


With advancements in treatments for Wolfram syndrome come new ways to possibly treat or further understand other degenerative diseases such as Alzheimer’s and Parkinson’s. There are multiple forms of treatments being devised, one path is to limit the frequency of cell death and halt further symptoms the other to prevent or replace the mutated cells. Treatments to limit cell death include clinical trials conducted with Dantrolene which helps to preserve cells that may be suffering from ER stress. Another treatment is the use of sodium valproate which keeps cells with defective wolframin from dying. Gene therapy is a new treatment that has yield promising results as well as expanding the knowledge on other degenerative diseases. Researchers have also employed the use of gene editing through CRISPR to determine if the defects can be located and amended to eliminate the mutation. With the new gene-editing data researchers have found that when corrected stem cells are developed into beta cells and placed back into mice suffering from diabetes their blood sugar returned to normal. Researchers are working on implementing this technique to develop healthy wolfram genes. With all these new discoveries and advancements with Wolfram Syndrome other similar degenerative diseases will be easier to study. The main issues that confront further progress are funding and limits to our current scientific understanding and tools.

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