Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene.
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W. Low | R. McIvor | C. Whitley | R. Gunther | D. Pan | P. Graupman | Joel L. Frandsen | B. Koniar | S. Hartung | Brenda L. Koniar