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Neurogenetic paper
According to a new case study published in the journal Nature Medicine, nearly 40 years ago, a blind person diagnosed with retinitis pigmentosa (RP) received a new type of photogene therapy and partially recovered his vision. The result of this medical research is the first report on the functional recovery of patients with neurodegenerative diseases after phototherapy. Photogene therapy involves genetically modifying specific cells to respond to light pulses, and then controlling these cells. .

According to this paper, retinitis pigmentosa is a neurodegenerative eye disease, which will destroy the photoreceptor cells in the retina and may lead to complete blindness. At present, there is no other approved treatment for retinitis pigmentosa except gene replacement therapy, which is only effective in the early stage of the disease. .

Correspondents Jose Sahel, Botond Roska and colleagues from the University of Pittsburgh described the preliminary results of the ongoing 1/2A phase study. In this study, they injected adeno-associated virus vector encoding photoreceptor christonr (for adjuvant gene therapy) into one eye of a 58-year-old blind person with retinitis pigmentosa, and used designed goggles to stimulate light. This kind of goggles is equipped with a special camera, which can convert the image of the visual world into light pulses, and then project the light pulses to the retina in real time to activate the transformed cells to complete the visual task. .

This previously blind patient has a good tolerance for this treatment. He wears goggles that can stimulate light, and can identify, count, locate and touch different objects with his treated eyes. The author thinks that phototherapy may help to restore the visual function of patients with retinitis pigmentosa. However, they warned that this experiment needs more results to further understand the safety and effectiveness of this method. . .