On Sep. 10, 2024, researchers at the Karolinska Institute in Sweden announced a study that shows that gene therapy can improve patients with Bothnia dystrophy, a form of hereditary blindness. Bothnia dystrophy occurs mainly in the region Västerbotten in Sweden, but the disease has also been identified in other parts of the world. The disease leads to progressive visual impairment due to the destruction of the visual cells in the retina.

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 preliminary results of the study show that the visual function of 11 of the subjects improved significantly. The study was published in Nature Communications.