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We rescued 1-week-old Mmut p.L690Ins/p.L690Ins mice with a single intraperitoneal injection of 3.02 10 10 vector genome copies per pup of an adeno-associated virus (AAV) vector expressing the wild-type human MMUT gene (AAV8.CB7.hMMUT) (37)

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Weeks 1-4: 2mg once weekly Weeks 5-8: 4mg once weekly Weeks 9-12: 8mg once weekly Week 13 onward: 12mg once weekly (maintenance) Total escalation time spans 12 weeks, slower than some competitors but necessary given the triple-receptor mechanism

The peptide preserved retinal morphological structure, intensified bioelectric activity, and improved functional capacity of photoreceptor cells