Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations
نویسندگان
چکیده
منابع مشابه
Postretinal Structure and Function in Severe Congenital Photoreceptor Blindness Caused by Mutations in the GUCY2D Gene
Purpose To examine how severe congenital blindness resulting from mutations of the GUCY2D gene alters brain structure and function, and to relate these findings to the notable preservation of retinal architecture in this form of Leber congenital amaurosis (LCA). Methods Six GUCY2D-LCA patients (ages 20-46) were studied with optical coherence tomography of the retina and multimodal magnetic re...
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ژورنال
عنوان ژورنال: iScience
سال: 2021
ISSN: 2589-0042
DOI: 10.1016/j.isci.2021.102409