Out of the Darkness, Into the Light With Gene Therapy

Updated March 14, 2019

There are a number of age-related diseases that affect people 50 and over. One such condition is age-related macular degeneration (AMD), which is the number one cause of vision loss in the United States. In the UK, it affects approximately 600,000 people, but researchers there are taking great strides to try to combat the disease with gene therapy.

How AMD Affects Sight

The macula in the eye is responsible for specific elements of your sight, notably the ability to see fine detail and central vision. When AMD sets in, it's because the cells in the retina are dying and not renewing, leading to loss of sight. There are two forms: wet and dry. Dry AMD is slow-progressing and a person's sight typically declines gradually over a period of several years. Wet AMD, on the other hand, is quick to develop and vision loss occurs much faster.

Clinical Trial Underway

According to an article by the BBC, an 80-year-old Oxford woman was the first patient to undergo gene therapy to see if there's a way to halt the process of vision loss. It won't be able to restore what has already been lost, but its aim is to stop the decline.

The gene therapy consists of an injection of a harmless virus containing the gene into the back of a person's eye. This virus then "infects" the retina, releasing the gene so it can get to work making a protein that stops the body's immune system from attacking the retina.

The clinical trial is in its early stages, and right now it's just a matter of testing whether the procedure is safe enough to proceed. The participants in the study already have a certain degree of vision loss. If it's effective in the long run, it could potentially treat patients with AMD before they lose their eyesight, and become a real game-changer in the medical field.

The team responsible for this clinical trial has already created another gene therapy treatment that's proven to be successful in treating choroideremia, another cause of vision loss, back in 2016, so hopes are high for success in this trial as well.

In 2010, the number of Americans estimated to have the disease sat at just under 2 million. The National Eye Institute estimates that by 2030, the number of those afflicted will rise to approximately 3.5 million. Gene therapy could prove to be the catalyst in the disorder, either slowing down vision loss due to AMD or putting an end to it altogether, proving once and for all just how amazing genomic research can truly be.

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