Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice

被引：0
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作者：
Carmen Agustín-Pavón
Michal Mielcarek
Mireia Garriga-Canut
Mark Isalan
机构：
[1] Imperial College London,Department of Life Sciences
[2] Cell and Developmental Biology Program,Current address: Predepartmental Unit of Medicine, Faculty of Health Sciences
[3] Centre for Genomic Regulation (CRG),undefined
[4] Universitat Pompeu Fabra (UPF),undefined
[5] University Jaume I,undefined
来源：
Molecular Neurodegeneration | / 11卷
关键词：
Monogenetic disease; Gene therapy; Huntington’s disease; Neurodegenerative disorder; Immune response; Synthetic transcription factors; rAAV; Host optimization;
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