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Gene Therapy for Choroideremia

by Scott Krzywonos, Thomas L. Edwards, MBBS, PhD (Cantab), FRANZCO

Tom L. Edwards, MBBS, PhD, FRANZCO, of the Nuffield Laboratory for Ophthalmology at the University of Oxford, England, reviews his research team’s approach to using gene therapy to treat choroideremia. Dr. Edwards summaries the basics behind choroideremia, explains the unique challenges of viral vector delivery to the retina, and discusses a phase 1/2 trial employing a gene therapy strategy to deliver treatment to the back of the eye.

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Comments (1)

VERY INFORMATIVE. THOUGHT OF SHARING MY CASE SERIES IN A FAMILY WHERE MOTHER AND DAUGHTERS (AGE 23 ADN 32) THOUGHT ASYMPTOMATIC BUT SHOW SIGNS OF CHOROIDERIMIA AND THE MOTHER HAS TYPICAL ISLANDS ON Fundus AUTOFLORESCENCE. MY QUESTION IS ABOUT THE COST AND SOME MORE INFORMATION REGARDS TREATMENT AND ITS PROTOCOLS. THANKS

murali (29 months ago)