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3. Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy. Audouard E; Khefif N; Mansat C; Nelcha O; Banchi EG; Lupiet C; Farabos D; Lamaziere A; Sevin C; Piguet F Mol Ther Methods Clin Dev; 2024 Jun; 32(2):101248. PubMed ID: 38680552 [TBL] [Abstract][Full Text] [Related]
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