Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient with RPE65Associated Leber’s Congenital Amaurosis

Rehan M. Hussain, Kimberly D. Tran, Audina M. Berrocal, Albert M. Maguire

Research output: Contribution to journalArticle

1 Scopus citations


Leber’s congenital amaurosis (LCA) is a rare inherited retinal degeneration (IRD) that causes severe vision loss, nyctalopia, and nystagmus within the first few years of life. RPE65 gene mutations cause approximately 6% of LCA cases and have become the target for therapy since voretigene neparvovec-rzyl became the first U.S. Food and Drug Administrationapproved gene therapy product for IRDs in 2017. The surgery involves pars plana vitrectomy with subretinal injection of a viral vector that carries a functional copy of the RPE65 gene. Intraoperative optical coherence tomography is a useful adjunctive tool to confirm the injection has reached the subretinal space.

Original languageEnglish (US)
Pages (from-to)661-663
Number of pages3
JournalOphthalmic Surgery Lasers and Imaging Retina
Issue number10
StatePublished - Jan 1 2019
Externally publishedYes


ASJC Scopus subject areas

  • Surgery
  • Ophthalmology

Cite this