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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial - The Lancet
A Novel Mutation in the RPE65 Gene Causing Leber Congenital Amaurosis and Its Transcriptional Expression In Vitro | PLOS ONE
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Luxturna: A Gene Therapy Reviving Retinal Cells in Children with Leber Congenital Amaurosis | Arizona RETINA Project
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Overview of RPE65 mutations reported to date in the Chinese population... | Download Scientific Diagram
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IJMS | Free Full-Text | An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials
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Cells | Free Full-Text | The Predicted Splicing Variant c.11+5G>A in RPE65 Leads to a Reduction in mRNA Expression in a Cell-Specific Manner
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RPE65-related retinal dystrophy: mutational and phenotypic spectrum in 45 affected patients | medRxiv
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Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis: Molecular Therapy
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Schematic of the gene structure of RPE65 showing the positions of the... | Download Scientific Diagram
![A Gene Scan Study of RPE65 in Chinese Patients with Leber Congenital Amaurosis | Chinese Medical Journal A Gene Scan Study of RPE65 in Chinese Patients with Leber Congenital Amaurosis | Chinese Medical Journal](https://mednexus.org/cms/10.4103/0366-6999.218007/asset/13a82100-1d09-4159-83c1-cc741f145d06/assets/graphic/0366-6999-130-22-111-f002.png)
A Gene Scan Study of RPE65 in Chinese Patients with Leber Congenital Amaurosis | Chinese Medical Journal
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