This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA.
Name: rAAV2-ND4
Description: a Single IVT InjectionType: DrugrAAV2-ND4
Description: The Best Corrected Visual Acuity
Measure: BCVA Time: Change from Baseline at 12 months .Description: visual evoked potential
Measure: VEP Time: Change from Baseline at 12 months .Description: retinal nerve fiber layer
Measure: RNFL Time: Change from Baseline at 12 months .Single Group Assignment
There is one SNP
Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic Neuropathy This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA. --- G11778A ---
This is a multi - center , prospective study of 142 patients with the G11778A mutation in Mt-DNA . --- G11778A ---