Article
Voretigene Neparvovec for the Treatment of RPE65-associated Retinal Dystrophy: Consensus and Recommendations from the Korea RPE65-IRD Consensus Paper Committee.
Korean journal of ophthalmology : KJO - 1 Apr 2023
Han Jinu, Joo Kwangsic, Kim Ungsoo Samuel, Woo Se Joon, Lee Eun Kyoung, Lee Joo Yong, Park Tae Kwann, Kim Sang Jin, Byeon Suk Ho
Abstract excerpt
Mutations in the RPE65 gene, associated with Leber congenital amaurosis, early-onset severe retinal dystrophy, and retinitis pigmentosa, gained growing attention since gene therapy for patients with RPE65-associated retinal dystrophy is available in clinical practice. RPE65 gene accounts for a very small proportion of patients with inherited retinal degeneration, especially Asian patients. Because...
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