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Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy.
Mol Ther Methods Clin Dev. 2024 Apr 6;32(2):101248. doi: 10.1016/j.omtm.2024.101248. eCollection 2024 Jun 13.
Mol Ther Methods Clin Dev. 2024.
PMID: 38680552
Free PMC article.
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