Treatment of Leber Hereditary Optic Neuropathy with G11778A Mitochondrial DNA Mutation
Patients with Leber hereditary optic neuropathy (LHON) who carry the G11778A mitochondrial DNA mutation represent a genetically defined sub-population for which targeted therapeutic investigation has been conducted in multiple clinical trials.
Clinical scenario: LHON confirmed in the context of the G11778A mitochondrial DNA mutation — the specific genetic variant addressed by this first-line protocol.
Treatment approach
For this genetically defined form of LHON, the protocol involves a targeted gene therapy intervention. The complete regimen — including administration details, eligibility criteria, and clinical trial considerations — is available in the full protocol.
References
- Multiple recent clinical trials have investigated treating LHON patients who have the G11778A mutation with a one-time intravitreal injection of viral-mediated gene therapy.
- Gene therapy is currently only available within the context of a clinical trial, unless special approval is sought.
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