Gene Therapy and the Greatest Living Guitarist

In last week's blog post, I discussed the use of gene therapy as a treatment, and possible cure, for Parkinson's disease. At the end of the post, I mention my wish of seeing Muhammad Ali, who I refer to in the post title as the 'greatest boxer of all time,' cured of Parkinson's tremors. Well, I couldn't help but follow up on that post when I saw a brief article in Popular Science about an 8-year-old boy whose eyesight is restored in an eye treated with gene therapy. According to the original article in The Lancet, patients of the study were treated with a vector that encoded a protein that controls the activity of the retinal pigment epithelium, restoring eyesight in six of the twelve patients in the study with congenital blindness. The results indicate that, by studying both children and adults, the greatest improvement in eyesight is achieved when patients are treated with gene therapy at a young age, as the children of the study fared better than the adults.

Despite being somewhat void of details, the Popular Science article did have something that the original Lancet article did not: a video of the 8-year-old boy navigating a maze first using only his control eye, which did not undergo gene therapy injections, and then using his treated eye, which did. The difference is quite obvious. In fact, the first video of the boy trying to navigate the maze using his control eye was somewhat difficult to watch, as he had to be assisted at every turn. He even struggled to find the door knob on the door at the end of the maze. On the other hand, the video of his attempt using the treated eye is much easier to watch. Let's just say, if it had been a test, he would've aced it.

Very cool study and very promising results.

"But what's with the weird title" you ask? As I stated earlier, last week's blog post title makes reference to Muhammad Ali as the 'greatest boxer of all time,' and since I'm following up on that post, I thought I'd continue the theme. For those of you not in the know, the 'greatest living guitarist' refers to Jose Feliciano, who was born permanently blind. At 64 years of age, it's unlikely that Feliciano's vision will ever be restored with gene therapy, but it's appealing to think of a future in which congenital blindness has become a thing of the past.