AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy.

Mol Ther · 2014 · PMC3944324 · PMID 24162799

Europe PMC DOI

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highly significantno p-value reported

At high dose (3 × 10 10 vg), both CMV and tMCK vectors produced statistically highly significant improvements in CMAP amplitudes compared to PBS control group.

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