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Subretinal deposits in young patients treated with voretigene neparvovec-rzyl for RPE65-mediated retinal dystrophy
  1. Jennifer Lopez1,2,
  2. Mark Borchert1,2,
  3. Thomas C Lee1,2,
  4. Aaron Nagiel1,2
  1. 1The Vision Center, Department of Surgery, Children's Hospital Los Angeles, Los Angeles, California, USA
  2. 2Department of Ophthalmology, USC Roski Eye Institute, Keck School of Medicine, Los Angeles, California, USA
  1. Correspondence to Dr Aaron Nagiel, The Vision Center, Department of Surgery, Children's Hospital Los Angeles, Los Angeles, California, USA; anagiel{at}chla.usc.edu

Abstract

We report a series of three young patients (ages: 22 months, 2 years, and 5 years) who developed subretinal deposits at post-operative week one following subretinal voretigene neparvovec-rzyl treatment for RPE65-mediated retinal dystrophy. In the 5-year-old, subretinal deposits were also observed in the inferior periphery of both eyes. All three patients experienced improved visual function with treatment, and both the macular and inferior subretinal deposits have improved or resolved over the follow-up period. These findings may inform the delivery parameters and safety profile of AAV-based gene therapy as the number of retinal gene therapy trials continues to grow.

  • Retina
  • Degeneration
  • Treatment Surgery

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Footnotes

  • Competing interests AN serves as a paid consultant for Atsena, Eyebiotech, Janssen, Novartis, and Biogen.

  • Provenance and peer review Commissioned; externally peer reviewed.