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2. Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and preliminary evidence for a biochemical effect

Author

Flanigan, Kevin M., primary, Truxal, Kristen V., additional, McBride, Kim L., additional, McNally, Kelly A., additional, Kunkler, Krista L., additional, Zumberge, Nicholas A., additional, Martin, Lisa, additional, Aylward, Shawn C., additional, Corridore, Marco, additional, McKee, Christopher, additional, Fu, Haiyan, additional, and McCarty, Douglas M., additional

Published
2017
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