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Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retina.

Mol Ther · 2025 · PMC12265955 · PMID 39489914

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Oat −/− animals treated only by liver-gene therapy showed a variable reduction of intraocular ornithine levels compared to affected untreated eyes 6 months post-administration that did not reach statistical significance ( Figure 2 B).

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