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A Novel Small <i>NPC1</i> Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease by Michael Paul Hughes, Hemanth Ramesh Nelvagal, Oliver Coombe-Tennant, Dave Smith, Claire Smith, Giulia Massaro, Laura Poupon-Bejuit, Frances Mary Platt, Ahad Abdul Rahim
Published 2023-06-01
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