Gene Therapy at Kellogg Eye Center – Zion’s Story

(soft music) – Zion was born in Tennessee. At three months old we realized he was not acting like a normal child. So we got him into a specialist and he was diagnosed with
Leber congenital amaurosis. About six months ago, he had gene replacement therapy, LUXTURNA. – Zion is one of our very first patients who have received LUXTURNA treatment at the Kellogg Eye Center. And once the gene
therapy became available, we discussed it with
the family and with Zion and they were very excited to have this as a treatment option. – [Daisy] We are hoping that
that will improve his vision for as long as possible. – Generally, the job of a
gene is to produce a protein. And proteins have lots
of different functions within our eyes. Some will help with structure, some will help different reactions occur, and if you have a genetic mutation, or a genetic change that makes
the gene not work properly, than the proper protein is not produced. – [Cagri] For the gene therapy
the treatment is typically administered with surgery, and the other option is to do an injection directly into the eye. So what we do is we
deliver the normal copy of the gene under the retina
and targeting the cells that have the dysfunctional
copy of the gene. The treatment requires the treatment of each eye individually. So we’re typically doing two surgeries on two different days. Our hope is that we will be able to stop the degeneration in his retinas and preserve his visual function for many, many years to come. – [Diana] We’re hoping, it’s supposed to, make it to where if he
needed this much light to see in the first place, you know, with the drug he only
needs this much light. You know what I mean? To make a difference. So, that’s what the hope is. We’ll see how it goes. (digital music) – [Cagri] Patients like Zion who have Leber congenital amaurosis have night blindness. So they have a very difficult
time functioning in dark. And one of the first treatment effects we’re noticing in our patients after they receive LUXTURNA is that they’re telling us
they’re doing much better in dark, so they’re able
to stay outdoors longer. They’re also able to
navigate around the house. They don’t need night lights anymore. And then they’re able to participate in indoor sports. So that’s been a huge
change in their life. – [Kari] And so we hope
that you know, as Zion, we’ve kinda changed
the tragectory for him. To a point where, when he’s in his 20’s he won’t have as
significant of vision loss as what we’re seeing with him now.

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