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Ermira Samara-Kuko

Free University of Brussels

16

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264

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piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts

2016 Nucleic Acids Research
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Next-generation muscle-directed gene therapy using skeletal-muscle specific transcriptional modules identified by genome-wide computational analysis

2015 Human Gene Therapy
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Erratum: Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy (Molecular Therapy (2014) 22 (1614-1624) DOI:10.1038/mt.2014.131)

2015
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Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

2014 Molecular Therapy
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Hepatic Delivery of Genes Encoding Therapeutic Proteins or Mirnas Using Hyperactive Transposons: A Platform Technology for Gene Therapy and Functional Genomics In Vivo

2013 Molecular Therapy
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Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

2011 Hepatology
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"Taming" iPS: Coaxed differentiation of iPS into myogenic precursors using hyperactive transposons expressing the myogenic transcription factor PAX3

2010 Human Gene Therapy
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Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation

2010 Stem Cells
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Efficiency of stable transduction with integration-defective lentiviral vectors is tissue-dependent

2008 Human Gene Therapy
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Novel enhanced transposases result in robust stable gene transfer into hematopoietic, mesenchymal and muscle progenitor/stem cells

2008 Human Gene Therapy
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