Advances in gene therapy for Wolfram syndrome
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DOI: 10.1111/j.1755-3768.2017.03174
Abstract: Summary The Wolfram Syndrome (WS) is an early onset genetic disease (1/200 000) featuring diabetes mellitus and progressive optic neuropathy ensuing mutations in the WFS1 gene. To date, there is no treatment to stop the… read more here.
Keywords: therapy wolfram; gene therapy; wolfram syndrome; gene ... See more keywords