Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I by Lalitha R. Belur, Avery K. Huber, Hillary Mantone, Mason Robertson, Miles C. Smith, Andrea D. Karlen, Kelley F. Kitto, Li Ou, Chester B. Whitley, Elizabeth Braunlin, Justin Furcich, Troy C. Lund, Davis Seelig, Carolyn A. Fairbanks, Nicholas Buss, Kwi Hye Kim, R. Scott McIvor

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