Author

Ahmad Al Saneh

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Open access Jul 2026

Programmed repair of disease-causing UGA premature termination codons in mammalian brain

Data demonstrate that U6 promoter-driven and single-stranded AAV2/9 constructs show variable and dose-dependent activity, whereas self-complementary AAV2/9 with the tRNA in a minimal 100-bp genomic context provides broad and efficacious PTC rescue.

Ahmad Al Saneh, Lionel Gissot, Christopher A. Ahern · Nucleic Acids Research · 0 citations