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AAV design for gene replacement

Posted 2023-12-27 07:20:13

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AAV design for gene replacement

In gene replacement therapy, the AAV delivery system is critical to the expression of the transgene in target cells. Currently, AAV-based gene replacement strategies have been developed to treat retinal degeneration. The results indicate that AAV plays an important role in repairing retinal function and photoreceptor structure in a wide collection of animal models, such as mice, rats.

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