Hughes, Michael Paul;
Nelvagal, Hemanth Ramesh;
Coombe-Tennant, Oliver;
Smith, Dave;
Smith, Claire;
Massaro, Giulia;
Poupon-Bejuit, Laura;
... Rahim, Ahad Abdul; + view all
(2023)
A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease.
Cells
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