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 | |
| State | Published - Jan 1 2019 |
| Externally published | Yes |
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ASJC Scopus subject areas
- Surgery
- Ophthalmology
Cite this
Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient with RPE65Associated Leber’s Congenital Amaurosis. / Hussain, Rehan M.; Tran, Kimberly D.; Berrocal, Audina M.; Maguire, Albert M.
In: Ophthalmic Surgery Lasers and Imaging Retina, Vol. 50, No. 10, 01.01.2019, p. 661-663.Research output: Contribution to journal › Article
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TY - JOUR
T1 - Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient with RPE65Associated Leber’s Congenital Amaurosis
AU - Hussain, Rehan M.
AU - Tran, Kimberly D.
AU - Berrocal, Audina M.
AU - Maguire, Albert M.
PY - 2019/1/1
Y1 - 2019/1/1
N2 - 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.
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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UR - http://www.scopus.com/inward/citedby.url?scp=85074396422&partnerID=8YFLogxK
U2 - 10.3928/23258160-20191009-01
DO - 10.3928/23258160-20191009-01
M3 - Article
C2 - 31671202
AN - SCOPUS:85074396422
VL - 50
SP - 661
EP - 663
JO - Ophthalmic Surgery Lasers and Imaging Retina
JF - Ophthalmic Surgery Lasers and Imaging Retina
SN - 2325-8160
IS - 10
ER -