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Gene therapy: Novel targets for congenital blindness

September 2, 2020

As many as 40,000 people in Germany suffer from retinitis pigmentosa. This hereditary disorder is characterized by loss of photoreceptors in the retina, and can be caused by mutations in many different genes. Depending on the nature of the underlying genetic defect, the severity of the condition can vary between night blindness and progressive visual field loss that can ultimately result in total blindness. The first gene therapies for the disease have recently been approved. However, these approaches have certain disadvantages, which limit their range of application.

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