[PERSPECTIVES] Leber Congenital Amaurosis Caused by Mutations in GUCY2D

This article will summarize clinical characterization of patients and proof of concept gene replacement studies in several animal models of GC1 deficiency, both of which have laid the groundwork for clinical application of a gene therapy for treatment of LCA1.

Source: Cold Spring Harbor perspectives in medicine - January 5, 2015 Category: Research Authors: Boye, S. E. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research