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An AAV Encoding Human ABCD1 Shows Dose-Responsive Expression and Function Following Spinal Cord Delivery in a Rodent Model of Adrenomyeloneuropathy

Honggeun Park, Tony del Rio,Vidyullatha Vasireddy, Tiffany Lutz,Carrie Ng, Yi Gong,Inge Dijkstra,Stephan Kemp,Casey Maguire,Sean Clark, David Anderson,Karen Kozarsky

NEUROLOGY（2022）

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