Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease

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Bibliographic Details
Published in:Molecular genetics and metabolism 2020-02, Vol.129 (2), p.S22-S23
Main Authors: Armour, Sean M., Nordin, Jayme M.L., Verdera, Helena Costa, Sellier, Pauline, Preston, George M., Riling, Christopher, Cohen, Daniel M., Collaud, Fanny, Crosariol, Marco, Quinn, William J., Hanby, Hayley, Cagin, Umut, Puzzo, Francesco, Haurigot, Virginia, Ronzitti, Giuseppe, Colella, Pasqualina, Anguela, Xavier M., Mingozzi, Federico
Format: Article
Language:English
Online Access:Get full text
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ISSN:1096-7192
DOI:10.1016/j.ymgme.2019.11.029