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Our research has initially focused on gene therapy solutions for severe inherited retinal diseases. In the longer term, we hope to find solutions for patients faced with neurodegenerative diseases more generally.
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  • Leber's Hereditary Optic Neuropathy
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  • Diseases we target
  • GS010 (lenadogene nolparvovec)
  • GS030
  • GS030 for Geographic Atrophy in Dry-AMD
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