In 2017, the FDA approved the sales of gene therapy treatment for inherited blindness and inherited eye diseases. While the treatment doesn’t completely cure blindness, it does allow patients to see shapes and lights so that they can get around and live normal lives without the aid of a cane of guide dog. It’s pretty incredible, and this is really only the beginning of the available applications for a huge variety of patients suffering from a huge variety of optical ailments.
Gene therapy is defined as “the use of genes to treat various medical conditions.” If that seems vague, it’s because it can be applied to almost anything, not just ophthalmology. The loose concept basically consists of transferring genetic materials into tissues, organs and cells with the aim of curing a disease or improving their clinical status. Ideally, the new genes from gene therapy will replace the faulty genes causing the illness and will encourage gradual genetic expression until they’re totally normal. Obviously, these breakthroughs have a wide range of applications, but they have opened more doors for the ophthalmology than for any others. This is mainly because there is a vector (type) of gene therapy that affects the retinal cells drastically. Because of this, we can now target a variety of diseases that are most commonly the causes of eye illnesses that cause serious defects like blindness.
Since a specific vector aids in healing gene mutations that cause blindness there has already been a huge surge of patients that are now slowly recovering from these diseases.
The perfect candidate recently arose in Florida. A boy by the name of Creed Pettit had a genetic condition called Leber congenital amaurosis, which trailed him through his young life with progressive darkness. HIs retina could not pick up light unless it was quite bright and so he had to adapt by carrying around flashlights so that he might be able to see the food in his lunch box at the least. Thanks to impressive applications of gene therapies though, he’s starting to see more closely to normal and will be able to live his life to the fullest, even in dim lighting. You can read more about Creed and the revolutionary treatment here.
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