Hypotheses: The primary hypothesis being tested is that there will be no toxicity resulting in loss of vision to no light perception in injected eyes.
Name: injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),
Description: injection of Total Volume of each intravitreal injection is 200 µLType: Drug1(Chronic) 2(Acute) 3(Presymptomatic)
Name: injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med)
Description: injection of Total Volume of each intravitreal injection is 200 µLType: Drug1(Chronic) 2(Acute) 3(Presymptomatic)
Name: injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)
Description: injection of Total Volume of each intravitreal injection is 100 µLType: Drug1(Chronic)
Name: injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)
Description: injection of Total Volume of each intravitreal injection is 100 µLType: Drug1(Chronic) 2(Acute) 3(Presymptomatic)
Description: Incidence of local and general adverse events and Serious Adverse Events
Measure: Assessment of Primary Endpoint - Toxicity Time: 1 yearAllocation: Non-Randomized
Parallel Assignment
There is one SNP
An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial DNA. --- G11778A ---
Inclusion Criteria: 1. Age 15 or older; 2. Patients with LHON and the G11778A mitochondrial DNA mutation. --- G11778A ---
A previous CLIA certified genetic lab result showing the LHON G11778A mutation will be accepted for inclusion; 3. Ability to perform tests of visual and retinal function; 4. Ability to comply with research procedures; 5. Able and willing to provide informed consent before undergoing any study related procedures. --- G11778A ---