Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

2009/10/24 by Albert M. Maguire, Albert M Maguire, Katherine A. High +45 · 5 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Ocular Disorders and Treatments #Retinal Development and Disorders #Retinal Diseases and Treatments

paper · doi:10.1016/s0140-6736(09)61836-5

openalex publication_date 2009/10/24 · crossref created 2009/10/24 · crossref issued 2009/11/01 · crossref published 2009/11/01 · crossref published-print 2009/11/01 · crossref deposited 2025/09/25 · openalex created_date 2025/10/10 · openalex updated_date 2026/08/01 · crossref indexed 2026/08/01

Citations

• Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year
• Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
• Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis
• Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
• Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
• Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

Cited by

• Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
• The pigmented epithelium, a bright partner against photoreceptor degeneration
• The Retinal Pigment Epithelium in Health and Disease
• The human visual cortex responds to gene therapy–mediated recovery of retinal function
• Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
• Retina [wikipedia]