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[DOI] [PubMed] [Google Scholar] Nguyen T.T., Emami F., Yook S., Nguyen H.T., Pham T.T., Pathak S., Regmi S., Kim J.O., Yong C.S., Kim J.R., Jeong J.H

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Its mechanisms of action are linked to its ability to bind copper and facilitate its biological functions

Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy
