The 2017 FDA approval of the first gene therapy for a genetic disease in the United States marked the beginning of a new chapter in the practice of medicine. This breakthrough has changed the future of counseling and management for patients with inherited retinal dystrophies, such as retinitis pigmentosa and Leber congenital amaurosis. As the field of gene therapy grows and as more patients qualify for available gene therapies and clinical trials, optometrists are needed to help with education, identification, and referral of patients who may benefit from the surge of innovative research and therapies.  This course presentation and discussion will cover the current state of ocular gene therapy and gene therapy research. Particular emphasis will be placed on FDA approved gene therapy eligibility and outcomes. We will provide an overview of current clinical trials, including developments in CRISPR gene editing and antisense oligonucleotide-based therapy. We will cover common patient questions and answers regarding gene therapy and genetic testing. We will address the current clinical guidelines for genetic testing and the incorporation of genetic testing into clinical practice. Patient and practitioner resources will be presented, including online patient registry and clinical trial resources.

Course Code:



Rachelle Lin, O.D.



AOA Expiration Date:


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