But I wanted to highlight a particular effort that addresses an important need in gene therapy for retinal degenerations: Delivering large corrective genes to cells in the retina.

For those of you who’ve seen the original “Jaws,” the summer blockbuster movie of 1975 about a killer shark terrorizing beachgoers, you may remember one dramatic scene. After he sees the enormous shark up close for the first time, Police Chief Brody, played by Roy Scheider, declares, “We’re gonna need a bigger boat.”

That’s the kind of situation we find ourselves in with diseases like Usher syndrome 2A (USH2A), LCA caused by defects in the CEP290 gene and RP caused by defects in EYS. We need a “bigger boat” to deliver healthy versions of these and other large genes to the retina. While current viral gene delivery systems, such as adeno-associated viruses and lentiviruses, are working well in clinical trials for retinal disease, they aren’t able to carry very large genes.

That’s where the nanoparticle-based gene therapy research being conducted by Dr. Muna Naash at Oklahoma University Health Sciences Center comes in. Nanoparticles are like tiny manmade rocks that are 1/12,000th the diameter of a human hair. Scientists can wrap just about any sized gene in them. Dr. Naash has shown that nanoparticles, with their therapeutic genetic cargo, are readily absorbed by retinal cells after being injected into the eye.

As part of this latest round of funding, we are supporting her development of a treatment for USH2A, but Dr. Nash’s emerging technology could be used to deliver large corrective genes for a variety of retinal diseases. So, we are excited about the potential for her treatment to help a lot of people.

Now, if you happen to be going to the beach soon, remember to wear sunglasses, sunscreen and a wide-brimmed hat – to protect your skin and your eyes. While they make for fun cinema, shark attacks are the least of your beachgoing worries, especially if “swimming” means you only go up to your ankles, like me.

Illustrated above: nanoparticles (courtesy of the National Institutes of Health)

In the study, researchers examined nearly 70 donor eyes, about half of which were affected by AMD. They ultimately identified 50 genes that may help predict AMD, and may also help scientists find new targets for treatments. That said, the investigators caution that it’s too early to draw any conclusions regarding all 50 genes and their roles in causing AMD and in the disease’s progression. They are focusing their efforts on 20 of what they believe are the more closely linked genes, but much more work needs to be done.

The following analogy might better explain what this finding means. Imagine there’s a city-wide network of crime rings. Based on some initial evidence, detectives identify and bring in 50 suspects for questioning. While rounding up these suspects is an important step in breaking up the crime network, much more investigation is needed to determine who plays an important role in the network and who doesn’t. More evidence needs to be collected to make arrests. Additional suspects need to be brought in for interrogation. The detectives also want to understand better how the crime network is organized, and how the different players interact with one another.

What I’m saying is, in many ways, scientific research is like trying to solve a really tough crime.

I think it’s also important to note that the Genome Medicine study confirms some important characteristics of AMD that many experts already suspect. Namely, that the condition is triggered, in part, by the innate immune system, the part of the immune system that fights infection and other foreign organisms. Scientists believe that damage to the retina occurs when the innate immune system fails to shut off at the appropriate time. Treatments are in development that target this disease pathway and a gene called Complement Factor H, which was first linked to AMD in 2005. So, again, we’re on the right path, but more work needs to be done.

Paul Simon might want to follow our lead here. While he’s one of the world’s most prolific and celebrated singer-songwriters, it appears he still has some research to do. He, indeed, claims there are “50 Ways to Leave Your Lover,” but he only mentions five in his song. Can you name the five?