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A “Bigger Boat” – The Role of Nanoparticle Treatments

NanoparticlesAfter a rigorous review process, the Foundation is funding eight new research projects for a wide range of conditions, including Leber congenital amaurosis (LCA), age-related macular degeneration (AMD) and retinitis pigmentosa (RP). You can read about these exciting projects in an article recently posted on the Foundation’s homepage.

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)

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6 Responses to 'A “Bigger Boat” – The Role of Nanoparticle Treatments'

  1. Bridgette says:

    Every article I have read on treatments and possible cures for rp and other eye diseases ae that they are very promising. Why are things not being done quicker. Is there that much red tape and bureacratic nonsense to keep you all from coming up with treatments for this. We have the most advanced people in this field working on things and everyone keeps saying promising promising . Do something before its too late for millions of people world wide. The technology with stem cells is there but everyone is scared of the words stem cells. Help these people now. Worry about the red tape later.

    • EyeOnTheCure says:


      Researchers are working as quickly and diligently as they can to make vision-saving treatments available. Good science – ensuring that a treatment is safe and effective – takes time. A typical clinical trial can take eight to ten years, and not every potential treatment will make it through the process. Whether we are trying to save vision, cure cancer, or prevent Alzheimer’s disease, success does not happen overnight. With that said, we are at an exciting juncture with more retinal treatment clinical trials underway – including those for gene therapies, stem cell treatments, and pharmaceuticals – than ever before. Five years ago, there were virtually no human studies in progress. And please keep in mind that the FDA plays a critical role in ensuring safety and efficacy. We are fortunate in this country to have the regulatory safeguards they provide.

      This blog post discusses the important Role of the FDA in clinical trials:

      Please feel free to call 1-800-683-5555 or e-mail if you would like to talk more about this issue.

      Thanks for your interest in our research.

    • RobertAZ says:

      Most of us; “blind people” very much appreciate the hard work involved with medical research. I also understand that research trials must be done safely and properly. Bridgette, may I suggest that you channel your frustration and anger to something positive; like raising much needed funds to speed-up some of the promising breakthroughs that FFB is a part of.

  2. Dan says:

    I have been reading “promising” articles for the last 10 years; praying, hoping and waiting. I think red tape is a much bigger boat.
    Stem cells are a blessing, we should take advantage…

  3. Pasha HAZRAT says:



    • EyeOnTheCure says:

      Dear Pasha, as you probably already know, Best disease, also known as vitelliform macular dystrophy, is an inherited form of macular degeneration characterized by a loss of central vision. For more information on Best disease, please see the following link:
      Excitingly, the Foundation Fighting Blindness is currently funding research efforts at the University of Pennsylvania and University of Wisconsin to develop a gene therapy treatment for Best disease. Already, preliminary data has been published showing that gene therapy can stop disease progression in a dog model. Here is the link: If successful, these research studies will be moved under the jurisdiction of FFB’s Clinical Research Institute which is focused on the development of human clinical trials. There is however, a lot of work to be done before a trial can start. Toxicity and dosing studies in large animals, additional safety studies, generation of a GMP viral vector that can be used in humans and the filing of regulatory paperwork with the FDA are just a few of the things that need to be done before the trial can start. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

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