Article

Single-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates

2025-08-02

Abstract excerpt

Adeno-associated viral (AAV) vectors are rapidly advancing as gene therapies for inherited and common retinal disorders, but gene therapy-associated uveitis (GTAU) limits their broader application. To investigate the primate ocular immune response, we administered subretinal AAV gene therapy to two non-human primates (NHPs): NHP1 received AAV2-CAG-hRPE65 (voretigene neparvovec) bilaterally at clinical dose; NHP2 r...

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Literature Corpus work: 8c215ffc-4e99-55cb-a3ef-cc3802559321
DOI: 10.1101/2025.07.31.667874

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