Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

2009/08/12 by Artur V. Cideciyan, William W. Hauswirth, Tomás S. Alemán +9 · 5 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Retinal Development and Disorders #Retinal Diseases and Treatments #Retinal and Optic Conditions

paper · pdf · doi:10.1056/nejmc0903652

openalex publication_date 2009/08/12 · openalex created_date 2016/06/24 · openalex updated_date 2026/07/28

Abstract

letter

Citations

• Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
• Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

Cited by

• Viral and nonviral delivery systems for gene delivery
• Gene therapy rescues cone function in congenital achromatopsia
• Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
• 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
• Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
• Gene therapy [wikipedia]