Reversing Blindness with New FDA Approved Retinal Gene Therapy

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Reversing Blindness with New FDA Approved Retinal Gene Therapy

In December 2017, the FDA approved a DNA altering medication that can reverse an inherited form of progressive blindness. Luxturna is a gene therapy which can treat a condition called Leber's Congenital Amaurosis (LCA) wherein people have inherited two faulty copies of the RPE65 gene. The therapy replaces those faulty genes with normal versions, thus erasing the mutations' harmful effects. A single injection in each eye has shown to be enough to improve lost vision. We are talking to the doctor couple, Dr. Jean Bennett of University of Pennsylvania and Dr. Albert M. Maguire of Childrens' Hospital of Philadelhia (CHOP), whose work for more than 25 years on congenital blindness led to this ground breaking FDA approval. Join us to learn about the novel therapy, details on treatment procedure and recovery.


Talk Recorded on June 12, 2018, 1 p.m. EST </> Embed

  • C June 12, 2018, 11:58 a.m.  US/Eastern

    I am so grateful for this groundbreaking technology and the people who have made this possible. I have a 10 year old daughter who is at her two week post op in one eye and one week post op on her other eye today! We have already noticed big gains in dim light, she is seeing things in a whole new light, and is sooooo excited about her new perspective. As a mom, to see her point to the sky and say “I see clouds”, nothing is more beautiful. Thank you, thank you, thank you! I can’t wait to see what the future holds for her now!

  • RS June 12, 2018, 9:21 a.m.  US/Eastern

    How can the current advanced status of developments towards cure for RPE65 and CEP290 leber's congenital amaurosis influence early-phase research initiated after identifications of new genes causing other inherited retinal diseases?

  • L June 12, 2018, 3:31 a.m.  US/Eastern

    Could a patient with other gene related diagnosis’ still be a candidate for retinal gene therapy

  • L June 12, 2018, 3:28 a.m.  US/Eastern

    What is the status of Cep290 Research and how close are we to a trial for mutations other than the c.2991+1655A>G Mutation?

  • A June 12, 2018, 1:20 a.m.  US/Eastern

    Does the virus used in the gene therapy treatment make you sick in any other way? (Does it do anything else to the body?)

  • P June 8, 2018, 7:53 a.m.  US/Eastern

    Can the same kind of approach be used to intervene in blindness conditions which are due to other mutations.

  • C June 8, 2018, 7:53 a.m.  US/Eastern

    How are the patients being selected to receive Luxturna? Is newborn screening plausible?

  • JC June 8, 2018, 7:43 a.m.  US/Eastern

    If it is seen that the vision is reducing again, do we do this procedure again?

  • TM June 8, 2018, 1:55 a.m.  US/Eastern

    Can this gene therapy science be applied to all of the other LCA disease causing genes?

  • PK June 5, 2018, 4:45 a.m.  US/Eastern

    Who can you treat - only children?

  • S June 5, 2018, 4:44 a.m.  US/Eastern

    Dr. Maguire, I heard this interview where you say that this thought of gene therapy came to you or started when you were in medical school and you discussed this with Dr. Bennett. Thanks for sharing.