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Genetic therapy helped children see

December 10, 2017

We are the edge of a world filled with incredible new advances in medicine thanks to a technique called gene therapy. Caroline and Cole, siblings from Arkansas, participated in a clinical trial in 2014 where they received a pioneering treatment using gene therapy that was able to restore their eyesight. Both children suffered from LCA prior to treatment.  Leber's congenital amaurosis (LCA) is a rare inherited eye disease that appears at birth or in the first few months of life and leads to blindness by adulthood.

The trial that used Luxturna product developed by Spark Therapeutics, was able to help children regain their vision. 

How did it work?

Gene therapy takes the approach of replacing faulty or missing DNA that causes diseases such as LCA among many others. A virus is used to infiltrate cells and deliver cloned normal DNA into the body. Eye is a perfect system for this due to the fact that it doesn't not have autoimmune cells on guard to fight any foreign cells/pathogens. Thus, virus can safely deliver it's content.

 

It's incredible to think that there are already gene therapy treatments that are being approved for cancer. Perhaps, in the near future, illnesses that were considered terminal will be able to be cured.

 

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