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

doi:10.3928/23258160-20191009-01

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

Ophthalmology

Research output: Contribution to journal › Article › peer-review

3 Scopus citations

Abstract

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 language	English (US)
Pages (from-to)	661-663
Number of pages	3
Journal	Ophthalmic Surgery Lasers and Imaging Retina
Volume	50
Issue number	10
DOIs	https://doi.org/10.3928/23258160-20191009-01
State	Published - 2019

ASJC Scopus subject areas

Surgery
Ophthalmology

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abstract = "Leber{\textquoteright}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.",

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AU - Maguire, Albert M.

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AB - 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.

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

Abstract

ASJC Scopus subject areas

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