Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates.

After administration, a decreasing trend in vector copies was observed in all NHPs by day 50 ( Figure 5 A).

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However, at 9 months, the viral vector copy number in the high-dose cohort showed a trend toward a decrease in all non-liver tissues ( Figure 7 A).

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