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Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice
Agustín Pavón, Carmen; Mielcarek, Michael; Isalan, Mark; Garriga-Canut, Mireia
Monogenetic disease
Gene therapy
Huntington’s disease
Neurodegenerative disorder
Immune response
Synthetic transcription factors
rAAV
Host optimization
© 2016 The Author(s). Open Access. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.
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