AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

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Authors: Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, Francesco Puzzo, Laetitia van Wittenberghe, Nicolas Guerchet, Nathalie Daniele, Bernard Gjata, Solenne Marmier, Severine Charles, Marcelo Simon Sola, Isabella Ragone, Christian Leborgne, Fanny Collaud, Federico Mingozzi

Journal title: Molecular Therapy - Methods & Clinical Development

Journal number: 12

Journal publisher: Cell Press

Published year: 2019

Published pages: 85-101

DOI identifier: 10.1016/j.omtm.2018.11.002

ISSN: 2329-0501