In the last several years, there have been significant advances in the area of targeted therapies for constitutional genetic disorders. In 2017, the FDA approved its first gene therapy, Luxturna, for the treatment of RPE65-associated retinal dystrophy. As a result of this breakthrough, gene therapy trials are underway for a variety of other retinal disorders. Retinal disorders exemplify the emerging paradigm that links molecular diagnostic testing for constitutional genetic disorders to targeted therapies.
Gene Therapy for Retinal Disorders
Elias Traboulsi, MD, MEd, Cleveland Clinic, Cleveland, OH, USA
Objectives:
- Describe novel targeted therapies for various genetic disorders.
- Describe the mechanisms of targeted therapies and patient prognosis.
Recording Date: November 20, 2020
Continuing Education Credit Information
CME/CMLE credit: 1.00 hr
Last day to purchase course and CE claim credit: February 16, 2024
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