RP1 Dual-AAV Gene Therapy Preserves Retinal Structure and Ameliorates Photoreceptor Degeneration in a Murine Model of Retinitis Pigmentosa.

چکیده

This study aimed to develop and compare dual-adeno-associated virus (AAV) gene therapy utilizing protein trans-splicing versus mRNA trans-splicing to restore RP1 expression, and to evaluate their safety and efficacy in rescuing photoreceptor degeneration in a mouse model of RP1-associated retinitis…

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