Wednesday, March 13, 2024

Altering the Human Genome

 A 10-year-old boy from Warren County, Jackson, was born with Leber’s Congenital Amaurosis (LCA) \ RPE65, he was treated with the first FDA approved gene therapy drug called Luxturna. This drug is provided to each eye and treats the genetic disease that causes blindness. This would add a working RPE65 gene in place of the mutated genes. With the new working gene, the cells would then begin to produce functioning protein. Jackson's distance vision started off as 20/1000 and after being treated with Luxturna his distance is no 20/80, a major difference and almost complete recovery of his vision. 

This story is crazy to me because I remember this kid from when I was in grade school. He was in preschool when I met him, way before he received this treatment. I had the pleasure of meeting him again years later, after he received the treatment, and he is a brand new person almost. Now, this once small kid in preschool, is in high school and can go about his daily life independently. It is truly remarkable what gene therapy can do, and I cannot wait to see what researchers can make of it in the future. 

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