Gene therapy for eye disease

Scientists have developed a new gene therapy approach that offers promise for one day treating an eye disease that leads to a progressive loss of vision and affects thousands of people across the globe. The study also has implications for a much wider suite of neurological disorders associated with aging dominant optic atrophy (DOA).

DOA is an inherited mitochondrial disease a neuro-ophthalmic condition characterized by a bilateral degeneration of the optic nerves causing gradual but subtle vision loss. DOA patients usually suffer moderate visual loss, associated with central or near central visual field deficits and color vision deficits.

Gene therapy works by:

  1. Scientist creating a new working copy of a missing or nonworking gene.
  2. The new gene is placed in a vector, which acts like an envelope that carries the gene to the right places.
  3. Next the vector is placed in the body and carries the new gene to the control center of the cells (Also known as the nucleus).
  4. Once inside the nucleus, the new gene tells the body how to make what it needs.
  5. The rest of the unused tractor is broken down by the body.

While still in pre-clinical trials researchers have found that a targeted gene therapy can treat DOA. The results so far demonstrate that this gene therapy can potentially provide benefits for a wider array of diseases involving mitochondrial dysfunction such as DOA.

Keep in mind this treatment is still in the trial phase and unavailable as treatment right now.







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