Gene therapy used for the first time to treat age-related macular degeneration

  • NIHR
  • 19 Feb 2019

  • curated by Pavankumar Kamat
  • UK Medical News
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Researchers at the University of Oxford in collaboration with the National Institute for Health Research have conducted the world’s first gene therapy operation for treating age-related macular degeneration (AMD).

AMD which affects more than 600,000 individuals in the United Kingdom is the most common reason for vision loss. The 'dry’ form of AMD slowly deteriorates the cells of the macula, significantly altering the central part of vision with gaps or ‘smudges’. Researchers hope that gene therapy may be able to halt the progression of AMD and preserve the remaining vision. In the future, it may be utilised in patients with early AMD to arrest the condition before vision loss commences.

The operation was performed on an 80-year-old woman from Oxford who had AMD in both eyes, with the left eye being afflicted to a greater extent. She faced significant difficulties with performing household activities, reading and recognising faces. The procedure involves detachment of the retina and injecting a virus-containing solution beneath the retinal surface. The virus carries a modified DNA sequence, which infects the retinal cells and corrects the AMD-causing genetic defect.

Professor Robert MacLaren from University of Oxford who led the study said: "A genetic treatment administered early on to preserve the vision in patients who would otherwise lose their sight would be a tremendous breakthrough and certainly something I hope to see in the near future."

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