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Breaking News: Positive Results for Second Eyes Treatment in Gene Therapy Trial

Drs. Jean Bennett and Albert Maguire

Drs. Albert Maguire and Jean Bennett lead the research team conducting the study.

I am very pleased to tell you that a Foundation-funded team at The Children’s Hospital of Philadelphia (CHOP) released positive results today regarding the treatment of the second eyes of three adult patients in an ongoing gene therapy trial for Leber congenital amaurosis (LCA). The results: After six months, it proved safe and improved light sensitivity, visual acuity and visual field.

So, what does this mean, exactly? Well, it’s the first time that patients in any retinal gene therapy clinical trials have received treatment in both eyes. And thanks to these successful results, the other nine patients in the CHOP study can now be invited to have their second eyes treated – which, I suspect, they’ve been looking forward to very much. This milestone, achieved by Drs. Jean Bennett and Albert Maguire (wife and husband, in case you didn’t know), is a big boost for the whole retinal gene therapy field, because it establishes a precedent for treating both eyes in human studies.

In fact, one other CHOP participant who has already received some second-eye treatment is Corey Haas, a boy whose first-eye success the Foundation reported on a few years ago. “Corey has had his second eye treated, and is doing well,” Dr. Bennett reports. “We are waiting to measure the results.”

All of this also bodes well for the eventual development of a treatment that can be made available to the public. And, by the way, there are four other LCA clinical trials out there. Collectively, more than 40 people have been successfully treated. In fact, researchers from the Universities of Pennsylvania and Florida recently published very nice three-year results for their clinical study.


35 Responses to 'Breaking News: Positive Results for Second Eyes Treatment in Gene Therapy Trial'

  1. RobertAZ says:

    Bravo! This news will surely bring many tears of joy. Congratulations to everyone involved. I believe this kind of treatment has the most promise IMO.

  2. Mr. Rose, do you have any information about how this can relate to forms of retinitis pigmentosa? Does this mean one day this type of therapy may be possible for RP patients? Being almost 30 years old now, diagnosed at the age of 12, it would be awesome if there was any positive gene therapy information out there for RP. Restoring vision type therapies. Again, any information is greatly appreciated. Love the blog by the way. It seems it’s going to be the best way for us impacted by a disease to get solid information. Keep it up!

  3. Suzanne Carver says:

    Thank you for providing funding, advocating and leading the way to finding cures for retinal diseases. The current research is very exciting and as a mom of a child born blind with LCA, the news is very encouraging.

    My 8 year old son has a defective GUCY2D gene. To my knowledge current studies are focused on the RPE65 gene. Are additional genes being studied and if not, is there a projection as to when they might?

    Suzanne D. Carver

  4. Betty Turner says:

    Thank you for sharing this fantastic news. We can’t wait to hear more of the details.

  5. S. Allen says:

    Granddaughter of almost 5 months is being seen for vision problems. As yet, there is no confirmed reason for the blindness. Optic nerves are said to be pink and healthy. Now the consideration is in the retina area. Where do I look for the experts??

    • Farshad says:

      Are they going to check for LCA mutations or other retinal disease mutations if the optic nerves are okay

      • EyeOnTheCure says:

        Diagnosing eye diseases in young children can be challenging. Obviously, kids have a harder time communicating their visual experiences. It is important to find a pediatric retinal specialist or ophthalmologist. Sometimes, it makes sense to consult with a neurologist, as well. Each case is different. Some research centers may have the expertise and resources to diagnose rarer conditions such as LCA or RP.

        I suggest you contact the Foundation at and we can try to help locate a doctor or facility in your area.

  6. Oran Hevlin says:

    Hi. My 5 years old son has LCA. I would love to hear
    More about this research.
    Could you send me more links?

  7. Richard says:

    Please can you let us know if the gene therapy can cure Stargardt macular dystrophy eye disease also.

    Thank you

  8. Edvaldo F de Castro says:

    I hope that Dr. Stephen Rose will shed some light on the current Clinical Trials of Stargen at The University of Oregon.

  9. Paul Smith says:

    Fantastic news!!! – I believe its only a matter of time before we unlock the potential of gene therapy for many congenital conditions …hopefully for patients with CXLRS also 🙂

    Well done – keep the faith people!

  10. Edvaldo F de Castro says:

    Will Dr. Rose write about the Stargen Clinical Trials at Oregon University ?

  11. Helena Ridge says:

    Anytime you see flashes of light you need to go see your eye doctor because it could be a retinal detachment. Also if you loose vision or it looks like a curtain is falling those are other symptoms of a retinal detachment. Please see your doctor right away.

  12. shaden says:

    Dear Sir
    I am from Saudi Arabia had sent earlier medical reports you
    I have two children girl the age of 4 years, and I’m two years old
    To have the disease in the cells of the retina of the cones
    Is there a cure?

  13. majid goussous says:

    thank you.
    i hope this will work for my 12 year old daughter.
    wish you all the best &happy holydays

  14. Reno says:

    My 18 Year old son has LHON. Are there any studies/trials currently being undertaken in Australia in relation to LHON treatment by stem cells. If so how can my son participate

    • EyeOnTheCure says:

      Reno, thanks for your comment. We would recommend that you visit We fund many clinical trials but do not operate the studies ourselves and so reaching out them directly may be the best way for you to find out more about where they are happening and if participating is an option.

      On, which is maintained by the National Health Institute in the U.S., you are able to search for clinical trials by searching based on your disease. Each clinical trial listing will tell you more about what the study is about and how you can contact them to see if they are recruiting and what the participant criteria may be.

      We hope this information is helpful and encourage you to contact us for additional help at for further information if needed.

  15. I have a daughter (avi soni 11yrs) diagonosised with LCA with flat erg .can she be treated?I am from Idar. gujarat. dist=sabarkantha. india .my cell phone=+919426337652.please help me. Thank you

    • Eye on the Cure says:

      Narayan, thanks for your comment. Currently, there is no treatment for LCA that has been deemed safe and approved for use, however much research is being done to find treatments.

      We have some information on our website about LCA here:

      We often also report out on new news on the research being done for this disease. Please see our blog articles on LCA here ( ), and be sure to check out our ‘other disease’ section on our website, where we often report on LCA research progress ( ).

  16. Hi my friend! I want to say that this post is awesome,
    nice written and include almost all imjportant infos.
    I would like to loopk extra posts like this .

  17. May god bring more improvements in gene therapy of lca clinical trail conducted at CHOP. wishing best of luck to dr .jean bennet & dr albert maguire for their success thank you

  18. Srinivas says:

    Hi All,

    Good to hear that there is a success case for LCA and I see a ray of hope for my daughter of 1.5 yrs old.
    She has retinal problem LCA due to GUCY2D.

    Could you please help is if there is any success or treatment for this. Please call me at +91 9849491203 if you come across any positive cases.

    Thank you,

  19. This a very good news for success ratio in Lca patients around the world. Best of luck. This has created good hope of vision for my 14yrs daughter diagonosised with Lca. Keep it up. Congratulations. My mobile no919426337652 thank you. narayan soni . India

  20. Shabaz Chagani says:

    Hi really exciting news,CONGRATULATIONS,I’ve been waiting to get a good news for my 2 kids also,they both have LCA and the mutated gene is rpgrip1.if any one has any information about it pls call me at 817-714 8386,my name is Shabaz Chagani,
    Thank you so much and God bless

  21. Shabaz Chagani says:

    Sorry I mistyped my phone number,right number is 817-714 8376
    Shabaz chagani

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