학술논문
Ocular gene therapy offers hope for inherited retinal disease: Nine pediatric patients with Leber congenital amaurosis had visual improvement.
Document Type
Article
Author
Source
Subject
*CLINICAL trials
*COLOR blindness
*EYE abnormalities
*GENE therapy
*PEDIATRICS
*RETINAL diseases
*USHER'S syndrome
*VIRUSES
*STARGARDT disease
*LEBER'S congenital amaurosis
*
*
*
*
*
*
*
*
*
Language
ISSN
0193-032X
Abstract
The article offers information on the treatment of inherited retinal disease in pediatric patients using ocular gene therapy, including choroideremia, usher syndrome, type B and Leber congenital amaurosis related to GUCY2D mutations. It mentions the first U.S. Food and Drug Administration-approved drug voretigene neparvovec-ryzl under the brand name Luxturna from Spark Therapeutics Inc. and other treatment option being explored called optogenetic gene therapy.