A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice.

Mol Ther Methods Clin Dev · 2024 · PMC11201152 · PMID 38934011

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showed a trendno p-value reported

The CPP16-MCOLN1-treated Mcoln1 −/− female group showed a trend toward higher rod retention in this test.

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