Combination gene therapy with AAV based RPE65 and survivin vectors sustains phenotypic rescue in neural retina of LCA2 mice

Key Points

• Phenotypic rescue in the neural retina of LCA2 mice indicates effective gene therapy outcomes.
• Combination therapy using AAV vectors led to improved visual function and retinal integrity.
• The research explores the synergistic effects of RPE65 and survivin gene delivery in an animal model.
• Findings highlight the importance of sustained therapeutic strategies in addressing genetic disorders.