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Empty AAV8 particles
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创建时间:
2025-05-14
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An efficacious and safe rescue of GM3 synthase deficiency in mice by spatially regulated AAV-mediated ST3GAL5 delivery. An efficacious and safe rescue of GM3 synthase deficiency in mice by spatially regulated AAV-mediated ST3GAL5 delivery
The first-generation rAAV-ST3GAL5 replacement vector employing a ubiquitous promoter led to hepatic injury and death caused by high off-target ST3GAL5 expression when administered systemically. In con
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