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Optogenetics: Seeing the Light in a Whole New Way

Video included
Image from optogenetics VideoAbout a year ago, I was blown away by a research paper that came across my desk by Dr. John Flannery, a Foundation-funded researcher at the University of California, Berkeley. In it, he reported how he’d restored some vision in mice that had no rods or cones. I recognized, right away, that this cutting-edge research had great implications for blind people.

Conventional wisdom says that if there are no rods and cones left, as is the case in late-stage retinitis pigmentosa, you’ll have no vision. And without replacing those rods and cones with new ones or an artificial device, vision restoration is simply not possible.

Here’s where John comes in. He accomplished his vision-restoring feat by using gene therapy to enable the ganglion cells in highly degenerated retinas to respond to light. Normally, ganglion cells aren’t light-sensitive; rods and cones are what convert light into the electrical signals sent to the brain, where they’re interpreted as vision.

Ganglion cells play a different role, providing the finishing touches to the visual information produced by rods and cones — fine-tuning it, if you will — before it makes its way to the brain. But in many retinal degenerations, ganglion cells survive long after rods and cones are gone, which is what makes them a prime target for vision restoration.

This new approach John and other investigators in research fields are taking is called optogenetics – a process in which gene therapy is used to empower cells, including those in the retina and the brain, to respond to light. In addition to restoring vision, scientists are exploring ways to use optogenetics to treat a variety of conditions, including Parkinson’s disease and sleep disorders. In fact, Nature magazine deemed it the “Method of the Year” in 2010.

The video below, produced by the McGovern Institute for Brain Research at MIT, does a nice job of explaining how optogenetics works and may, some day, restore vision in blind people.

In addition to supporting John, the Foundation is funding researchers in France and Switzerland to develop an optogenetic method to resurrect cones that have stopped working. Their goal is to launch a clinical trial of the treatment within three years. Also, FFB’s clinical arm, the National Neurovision Research Institute, is investing in RetroSense, a Michigan-based company, to bring optogenetics to the clinic. The company’s approach, like John Flannery’s, involves empowering ganglion cells for vision.

Throughout my career, I’ve had a lot of surprises – some good, some not so good. It’s the nature of scientific research. The important thing is to learn from failures and capitalize on successes. In the case of optogenetics, while there are still hurdles to overcome, we have an opportunity to restore vision in people with the most advanced vision loss. I am excited about the Foundation’s role in making it happen.


45 Responses to 'Optogenetics: Seeing the Light in a Whole New Way'

  1. RobertAZ says:

    This is an interesting approach. I guess we will find out if this light perception will result in usable vision or not in just a few years. Without the benefit of rods & cones; it’s hard to believe that Optogenetics will be able to recreate: depth, contrast and night vision. I’m no scientist, but this Optogenetics seems to merit more research JMO. MY hope is for the growth of new rods & cones with similar genetic therapy. IMO, genetics is the most exciting research on disease of all types and I do believe it is just a matter of time and research $ before cures are found.

    • meftah says:

      may be the ganglion are the God back up cone/rod system.
      I believe in all kind of therapies aproach stem cell, genetics, bionic, drug and optogenitic!!!

      I’m profoundly believer that within the decade, a new therapies generation will beworld wide spread and retoring sight will be commun cure.

  2. Dor says:

    Hello,

    I find this new research avenue an interesting and a very promising one.

    I wonder if that therapy is based on the identification of the defected gene and the replacement of it with a new correct one.
    May RP patients whose the defected gene has not been located yet be good candidates for such a procedure?

    Thanks,
    Dor

    • EyeOnTheCure says:

      We believe that patients (including those with RP) will not need a genetic diagnosis to receive optogenetic therapies. That’s one of the key advantages of this approach.

    • L says:

      It is not based on a specific defective gene. Most genetic causes of blindness are caused by proteins in the photorecptors. This therapy would target the bipolar cells and not be dependent on the specific cause of blindness. Therefore it should work equally well whether the specific gene that is the problem is known or unknown

  3. Carol AZ says:

    I have Retinitis Pigmentosa, this research sounds very promising.
    What is sad is that RP is inherited. There was nothing we could have
    done to prevent it. We are always looking (excuse the pun) for new ways
    to prevent or even cure it. You have no idea how much I pray for your
    research to help me and thousands of others.

    Thank you!!!

    • EyeOnTheCure says:

      Thanks for your support and feedback Carol! We are excited to have you as a reader :)

    • Roy Everett says:

      My brother (73), sister (55) and I (72) all have RP. We are all in late stages of the desease and hpve lost most of our vision. This is very interesting and i for one would volunteer for testing.

  4. Janey says:

    Good thought. I worry about the rods & cones not being part of the picture. I hope this works. Will the ganglion cells be strong enough to take on the job? I’ll be following this one.

    • EyeOnTheCure says:

      We are targeting ganglion cells because we believe they will be up for the job. That’s why they are one of our first targets for treatment. With that said, other cell types might work, as well. Resurrecting cones might be a good approach for some people. Stay tuned to these Foundation-funded efforts as we determine the best strategies for using optogenetics to restore vision.

  5. Katherine Michinaux says:

    Hi,

    I would like to know when the gen therapist will be available for stargard people? its any way that I can get in contact with the research labs or the doctor working on this? I sorry I just desperate my eye sight in getting worth every day and I can’t wait for a treatment or cure.

    Thanks

    Katherine

  6. Kristy O says:

    This brings great hope to my family! We all hope and pray and believe that there will be a cure to RP and other “vision robbing” diseases. And that my younger brother will have an amazing, bright future! Thank you for all your research and information!! WE are all extremely thankful!

  7. Although the text references the ganglion cells as the DNA target, the accompanying video indicates that the bipolar cells are the DNA target. As the bipolar cells are directly connected to the rods and cones and thus somewhat more “fine grained” with regard to the visual field, the potential for useful vision looks somewhat more promising.
    Am I grasping at straws to believe that this is a significant distinction, or are we really talking only about ganglion cells?

  8. Thomas says:

    This sounds great however we need a cure for AMD and other eye diseases now. What company is closest to restoring vision in patients?

    I look forward to your story on the success of Advanced Cell Technology’s current clinical trials here in the U.S. and UK for Stargardt’s and AMD

  9. Debbie Crosby says:

    Very interesting and informative. Much thanks to these scientists and to FFB for the research funding. Thats exactly the reason more people need to donate and participate in VisionWalk. I have great hopes that a cure will be found soon. I really enjoyed this. Thanks!

  10. Gary Paruta says:

    Attended the Vision Seminar in Florida on Saturday and really appreciated your presentation as it was very informative. But I have a special request regarding your future blog postings. I have RP and I found reading your blog rather difficult due to the colored print (green, blue, not sure) on top of green background. That creates a case of insufficient contrast for those of us who are visually impaired. And since your blog is intended predominantly for the visually impaired, would you mind putting dark print to white background in the future?
    Thank you.

  11. RobertAZ says:

    I was wondering if Optogenetics is the same therapy method I heard about on NPR a while back. I think that therapy used light to activate RNA, for a lack of a better term “switches” to affect a genes function. I’m pretty sure they were working with sleep disorder as well. I’m sorry; I don’t have a link to that story.

  12. Carlos says:

    If a patient receives gene therapy via an AAV vector to replace their known mutated gene, could they at a later time receive an AAV gene therapy to deliver an optogentic treatment to specific vision cells to make them light sensitive?

    Is there a limit on the number of times that a patient could potentially be administered an AAV gene therapy treatment?

    • EyeOnTheCure says:

      This is an interesting question:

      Two things:

      1) Researchers from Pennsylvania and Florida are already making multiple gene therapy injections to the same eye in LCA clinical trials. So, yes, preliminarily, it does look like multiple injections should not be a problem.

      2) However, if you would receive a corrective gene therapy (replacing bad gene with good gene), we would hope that treatment would prevent any further vision loss so you wouldn’t need optogenetic gene therapy. Would optogenetics and corrective therapies NEVER be used together? Hard to say right now. Maybe someday they could be used in tandem to maximize

  13. Marlow Lim says:

    Dear Researchers,

    Sincerely Thanks all of you for putting so much effort in the research looking to restoring the vision of those who suffers from RP and other eye diseases.
    Will do my little part in supporting the Foundation in every which way.

    Thanks again and God Bless.

    Marlow

  14. marcela avila says:

    My daughter has rp, she is 13 years and now she lost 90% of her vision. I hope this researches help my daughter and other to see.
    Thanks

  15. Lisa Evans says:

    This is fascinating news! My sister and I both have RP but while I still have sight, my sister doesn’t and she wants to be in a clinical trial very badly. I wish the researchers the best of luck! I will anxiously await more news on Optogenetics! Thanks for the info!

  16. Fran says:

    Thank you so much for this wonderful blog! This news is terrific. I have LCA caused by CEP290, I know with this mutation cones seem to be spared though they don’t work properly, so this might be the right way to go for me!

  17. Patty Wolverton says:

    Thanks for this information. It provides hope for those dealing with RP. My son is only 30 yrs old, had to give up his driver’s license a couple yrs ago, & has almost lost all peripheal vision. My question is whether or not to pursue genetics testing? I don’t have any history of RP on either my side or his Dad’s.

  18. Awesome I found that to be very interesting, thanks for sharing that! Can’t wait to read more!

  19. Foan Katrin says:

    Really nice article, really liked it. Read mine also here http://diseases-cure.blogspot.com.

  20. Amazing Webpage, Thx! Keep up the good work.

  21. Audre says:

    Fine, I’ll bookmark your blog, so I can come back and read more. Good job!

  22. RobertAZ says:

    After googling “optogenetics” I understand it alittle better. What I don’t understand is: Will the ganglion cells require an optic fiber to supply the right color light source to activate the cells or will the ganglion cells simply react to the natural light entering the eye? Thx.

  23. bathrooms says:

    Excellent article and easy to understand explanation. How do I go about getting permission to post part of the article in my upcoming news letter? Giving proper credit to you the author and link to the site would not be a problem.

  24. I agree completely. Thanks for the great information. I look foward to learning more. Have a great day!

  25. My son(18 yrs old) having deficiency of cones resulting in day blindness,v.poor vision & nystagmus.Your research has given me hope that my son can enjoy a normal life in future.
    Praying for more success,Best Regards.

    • EyeOnTheCure says:

      Farrukh, thanks for posting. We are glad that you find hope in our research updates. Like you, we are confident that one day this research will lead to a cure. We wish you and your family the best.

  26. Lou Scaltro says:

    My son is 27 years old and has XLRP (X-linked RP). Can Optogenetics be a potential therapy for XLRP?

    • EyeOnTheCure says:

      Lou,

      Optogenetics may ultimately benefit your son, especially if his disease is advanced. However, XLRP gene therapy, which we are funding, may be an even better approach for him. While neither approach is ready for human study just yet, it is important that you try to identify the genetic mutation causing his XLRP, if you haven’t done so already.

      Here is a Web link to information on genetic testing. We recommend that you share this with his retinal physician.

      http://www.blindness.org/genetic-testing

      If you have more questions about genetic testing or finding a retinal doctor who is familiar with XLRP, contact us at info@fightblindness.org.

  27. Linda Ringle says:

    My 6 year old grandson was diagnosed with LCA. The defective gene hasn’t been identified yet. Is this something that he could benefit from in the future? He sees some light if he’s outside on a sunny day.

    • jblasco says:

      Hi Linda,

      Optogenetics is for a person who has lost most or all of their photoreceptors, though it can also be used to resurrect cones (photoreceptors that provide central vision) that haven’t fully degenerated.

      Given that your grandson is only six years old, it is likely best if researchers continue to look for his disease-causing, perhaps re-testing his blood sample in another year or two as new genes and mutations are being discovered all the time. If they find his gene, then you can determine if a corrective gene therapy is emerging or available to treat his LCA. If his condition reaches a point where most or all of his photoreceptor are gone, then optogenetics might be a beneficial therapy.

      Please keep in mind that I am making some assumptions about the extent of your son’s disease. A doctor familiar with retinal degenerations and your son’s condition can make a better determination of what will benefit your grandson and at what point in his life.

  28. hanan mohamed raslan says:

    My husband has rp since 15 years l hope to find any treatment for him and he has anther two brothers have the same case he canont see now pleases send to me how cane abtain this treatment

  29. Mathias says:

    Anyone that can give me some info in swedish about optogenetic therapy or know if it exist in sweden? Or know where i can find info?

  30. pradeep meinam says:

    hello
    it’s a great news to hear that you have got the FDA clearance.I pray to god.hope some day it became the hope of many lives around the globe soon.
    thank you

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