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Source: Thailand Medical News  Aug 23, 2019  4 years, 6 months, 1 day, 17 hours, 10 minutes ago

New Gene Therapy For Age-Related Macular Degeneration (AMD)

New Gene Therapy For Age-Related Macular Degeneration (AMD)
Source: Thailand Medical News  Aug 23, 2019  4 years, 6 months, 1 day, 17 hours, 10 minutes ago
Dry age-related macular degeneration (AMD) is a common disorder that is caused by damage to the macula,the part of the eye that is responsible for our sharpest vision. It is the the leading cause of blindness and dry AMD, has no cure to date.

Dry AMD It can take years for signs of dry AMD to be found in the eye and often by the time it is diagnosed the disease is irreversible. Even if ophthalmologists find there are traces of dry AMD advancing in your retina there is nothing they can do for you, as your sight can not be saved. It is estimated that by 2020, there will be 198 million people living with AMD worldwide.

Fortunately, a new gene therapy being developed at The Australian National University (ANU) will help people at risk of dry and could potentially save millions of people from going blind. The team lead by Dr. Joshua Chu-Tan, from the John Curtin School of Medical Research, is developing a new eye injection that has the potential to stop the progression of the disease. And thus prevent the eventual blindness caused by the disease.

Dr Joshua Chu-Tan. Credit: Australian National University

Dr. Chu-Tan has been researching preventative gene therapy using tiny molecules called microRNA that he describes as "the kings of gene regulation." He has used microRNA to target the causes of AMD.

"We know inflammatory pathways  plays a major role in AMD and tiny molecules microRNA can fight them in a multi-pronged way. When we inject anti-inflammatory mircoRNA into the eye we see a decrease in genes responsible for inflammation and cell death, as well as a slowing in the damage progression of the retina.” commented Dr Chu-Tan in an interview with Thailand Medical News.

Dr. Chu-Tan said a specific microRNA, microRNA-124, had great promise as a preventative treatment for AMD as an alternative to the eye injections offered now. By injecting a cocktail of these molecules the researchers can slow the progression of this disease and hopefully halt vision loss.

When comparing an AMD patient to a healthy person, the healthy person has a lot of t microRNA-124 all over their retina, whereas in an AMD patient there is none. However it was noted that in the AMD patient's periphery of the retina, where there is no damage, there is microRNA-124.

The team is now planning various clinical trials simultaneously so as to hopefully get regulatory approvals as early as 2020 in order  to put the therapy out in the market.


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