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AAV-mediated gene therapy for sialidosis.

Mol Ther · 2024 · PMC11287007 · PMID 38796704

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Slightly increased NEU1 activity was measured in the brain stem, cortex, and cerebellum of the treated mice compared with the Neu1 −/− non-injected controls, although the values did not reach statistical significance ( Figure 2 A).

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