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ARVO 2018: Dr. Steve Rose Reports on CRISPR/Cas9 for Inherited Retinal Diseases

FFB’s own Dr. Steve Rose, chief scientific officer, reviews our commitment to funding and exploring CRISPR/Cas9 gene editing for inherited retinal disease in the video below.

FFB currently funds CRISPR/Cas9 projects at four institutions:

  • Johns Hopkins University (retinitis pigmentosa caused by the P23H mutation in RHO)
  • Columbia University (RP caused by the D190N mutation in RHO)
  • Massachusetts Eye and Ear Infirmary (RP caused by a mutation in RP1)
  • UCLA (Usher syndrome 1B caused by a mutation in MYO7A)

Want to learn more about the benefits of CRISPR/Cas9? Check out: A Cut-and-Paste Approach to Fixing Retinal-Disease Genes


16 Responses to 'ARVO 2018: Dr. Steve Rose Reports on CRISPR/Cas9 for Inherited Retinal Diseases'

  1. Gerale OBrien says:

    When will testing on humans began?
    How can we get into it? Is there an age limit?
    Thank you

    Also , I have a grand daughter with RETT syndrome. Any ideas on that area?
    Thanks again.

    • EyeOnTheCure says:

      Hi Gerale, these institutions’ CRISPR/Cas9 projects are not yet in clinical trials stages, but we will definitely be sharing when they are. And unfortunately our only area of expertise is in inherited retinal diseases. But best of luck!

  2. Has there been any research done on the CRB1 gene for Lebers congenital Amerosa?
    My granddaughter is 16 and has inherited Lebers congenital Amerosa.

  3. Rosamaria Somarriba says:

    My son is 44 years and has RP. Could he apply for a treatment

    • EyeOnTheCure says:

      Hi Rosamaria, at the moment none of the CRISPR projects are in the recruiting clinical trial stage, however, we recommend visiting http://www.clinicaltrials.gov and searching for other trials for RP that your son could participate in.

  4. Bridget Pacovsky says:

    Where are we with Stargardts? Making any advancement
    Thank you
    Bridget Pacovsky

    • EyeOnTheCure says:

      Hi Bridget, there are currently many ongoing clinical trials for Stargardt disease. Ophthotech has recently launched its Phase 2b clinical trial of Zimura® for people with Stargardt disease caused by mutations in the gene ABCA4. If you’re looking for other trials, we would recommend visiting https://clinicaltrials.gov/ and searching for recruiting trials for Stargardt disease.

  5. JO ANNE L. MENDES says:

    WHY WOULD MY EYELIDS START DROOPING

    I HAVE TO PULL UP MY EYE LIDS
    TO READ ANYTHING?
    IS THIS CAUSED BY MY RP?

    PLS HELP ME, I WANT TO WATCH MY
    GRAND CHILDREN GROW UP

    • EyeOnTheCure says:

      Hi Jo Anne, the Foundation is unfortunately not able to provide recommendations regarding medical care or treatments for a specific patient. We recommend you consult your doctor for your individual medical needs. Good luck!

  6. Marie Michele Del Balso says:

    Any cure for RP 73 (HGSNAT)

  7. Albert says:

    Dr Rose,
    I am 49 years old with RP and I am at the last stages of being completely blind I already lost sight in my right eye.
    When will clinical trials begin on humans?
    I would really like to be part of that.

    • EyeOnTheCure says:

      Hi Albert, there’s isn’t a set timeline for when the CRISPR/Cas9 projects will move into clinical trials. However, there are many other trials currently recruiting for RP. To learn more about clinical trials for RP, visit:
      http://www.clinicaltrials.gov. The site has a search function to find trials relevant to you. Good luck!

  8. Anthony Chapin says:

    Any improvements for retinal replacement or stem cells for Sticklers syndrome/ retinal detachment

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