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Invited Commentary
August 29, 2019

Gene Therapy for Choroideremia—Progress and Remaining Questions

Author Affiliations
  • 1Department of Ophthalmology, University of California, San Francisco
JAMA Ophthalmol. Published online August 29, 2019. doi:10.1001/jamaophthalmol.2019.3295

Inherited retinal degenerations, including choroideremia, have traditionally lacked safe and effective therapies because it was considered impossible to modify the consequences of genetic mutations that cause irreversible, progressive, photoreceptor death and blindness. However, clinical trials demonstrated the safety and efficacy of gene augmentation therapy for patients with inherited retinal degenerations due to mutations in the RPE65 gene,1 resulting in approval by the US Food and Drug Administration of the first gene therapy for an inherited disease in December 2017. That landmark development has renewed interest in development of gene augmentation therapies; currently, 23 different clinical trials are underway, studying gene augmentation for at least 13 different genes that cause inherited retinal degenerations.2

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