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Dose-response Evaluation of Intravenous Gene Therapy in a Symptomatic Mouse Model of Metachromatic Leukodystrophy

Emilie Audouard, Nicolas Khe, Charlotte Mansat, Oceane Nelcha, Elena-Gaia Banchi, Camille Lupiet,Dominique Farabos,Antonin Lamaziere,Caroline Sevin,Francoise Piguet

MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT（2024）

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关键词
Metachromatic leukodystrophy,gene therapy,intravenous,AAV
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