Gene Therapy Improves Vision in hereditary blindness: Study Finds
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Bothnia dystrophy is a form of hereditary blindness that leads to progressive visual impairment due to the destruction of the visual cells in the retina. A new study at Karolinska Institutet published in Nature Communications shows that gene therapy can improve vision in patients with the disease.
The disease is caused by an inherited genetic mutation that leads to damage to a particular protein in the eye. There is currently no treatment for the disease.
Researchers at the Karolinska Institute have now investigated whether gene therapy can improve vision in people with the disease.
The researchers used a so-called viral vector, a specially designed virus that was genetically modified to contain a functioning RLBP1 gene, the gene that is damaged in Bothnia dystrophy.
The viral vector was injected under the retina through an advanced surgical procedure in 12 people with the disease. The aim is that after treatment, the viral vector will be taken up by the cells of the retina, where it can produce normal protein.
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